This article was originally published here

Curr Opin Cardiol. 2021 Feb 16. doi: 10.1097/HCO.0000000000000841. Online ahead of print.

ABSTRACT

PURPOSE OF REVIEW: To highlight the evolving understanding of genetic variants, utility of genetic testing, and the selection of novel therapies for cardiac amyloidosis.

RECENT FINDINGS: The last decade has seen considerable progress in cardiac amyloidosis recognition given the advancement in cardiac imaging techniques and widespread availability of genetic testing. A significant shift in the understanding of a genetic basis for amyloidosis has led to the development of disease-modifying therapeutic strategies that improve survival.

SUMMARY: The systemic amyloidoses are disorders caused by extracellular deposition of misfolded amyloid fibrils in various organs. Immunoglobulin light-chain or transthyretin amyloidosis are the most common types associated with cardiac manifestations. Genetic testing plays a central role in the identification of genotypes that are associated with different clinical phenotypes and influence prognosis. Given the emergence of effective therapies, a systematic approach to the diagnosis of cardiac amyloidosis, with the elucidation of genotype when indicated, is essential to select the appropriate treatment.

PMID:33605615 | DOI:10.1097/HCO.0000000000000841

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Clinical approach to genetic testing in amyloid cardiomyopathy: from mechanism to effective therapies - DocWire News

Recommendation and review posted by Bethany Smith

SAN FRANCISCO, Feb. 24, 2021 /PRNewswire/ --Invitae Corporation (NYSE: NVTA), a leading medical genetics company, today announced the addition of tools from Medneon, a digital health AI company, to its robust clinical workflow and patient education tools. The additional capabilities further support clinicians and patients with cancer by making it easier to determine who should get testing and how to use genetic information to individualize treatment.

"While utility of genetic information in cancer care is well established, it remains challenging for clinicians to navigate varying guidelines and research findings that inform them about which patient, which test and what results mean for patient care," said Robert Nussbaum, M.D., chief medical officer at Invitae. "The addition of Medneon's risk assessment tool, which was developed by experts in the field, makes it easier for clinicians to make sure testing is considered and findings are acted upon for all patients who could benefit from genetic-informed care. The frequent updating of Medneon's recommendations based on the most current recommendations and newest research findings means that providers and patients will have the most up-to-date information at their disposal."

Medneon's Predictive Risk Assessment combines several factors, including current guidelines, real-world evidence, and personal and family history, to rapidly identify an individual's elevated short-term and lifetime cancer risk. Clinicians are quickly informed of their patient's eligibility for genetic testing and supplemental imaging, so that preventative action may be taken. In addition, the platform can assist clinicians in confirming and documenting the clinician's determination that the patient met medical necessity qualifying criteria to help navigate insurance coverage. When test results are returned, the company's Personalized Genetic Insights curates information from an array of published expert resources, including the AI knowledge base ASK2ME, medical experts, and genetic counselors, to generate custom reports that support patients and clinicians in shared decision-making.

"For clinicians who are learning to manage care through telemedicine during the ongoing pandemic, the Medneon platform distills the rapidly growing amount of published genetic and genomic information into digestible reports to help determine when to order a test, how to interpret results and how to personalize a care plan," said Noel Pugh, Ph.D., JD, MHA, head of commercialization at Medneon. "We hope by utilizing these tools, preventable cancers will be caught earlier to ultimately deliver better outcomes for patients and their families."

Medneon's technologies are another addition to the tools Invitae offers, anchored by its Gia platform, that support patients and providers by providing patient education and clinical support throughout the genetic testing process across a wide array of clinical areas.

About Medneon

Medneon's mission is to prevent cancer and optimize treatment by empowering individuals and their care team with DNA insights and decision support tools throughout the cancer journey. The innovative Medneon digital platform combines AI and human insights with actionable information regarding an individual's cancer risk to inform precision prevention and management over time at the point-of-care or through telemedicine. For more information, visitthe company's website at http://www.medneon.com.

About Invitae

Invitae Corporation(NYSE: NVTA) is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Safe Harbor Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the capabilities and potential benefits of the tools provided by Medneon. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company's ability to use rapidlychanging genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact:Laura D'Angelo[emailprotected](628) 213-3283

SOURCE Invitae Corporation

http://www.invitae.com

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Invitae adds Medneon's risk assessment tools to its education and clinical support offerings - PRNewswire

Recommendation and review posted by Bethany Smith

BEIJING, Feb. 24, 2021 /PRNewswire/ --Berry Oncology Corporation, the shareholding subsidiary of Berry Genomics, announced the results of a case-control study on the effectiveness of a novel diagnostic method for detecting hepatocellular carcinoma (HCC) in patients with liver cirrhosis, demonstrating the new model had greater accuracy and sensitivity compared to existing screening methods.

The study, published in Cell Research, featured both China and the U.S.-based authors and research teams led separately by Prof. Hong-Yang Wang of the National Center for Liver Cancer, Prof. Jin-Lin Hou of Nanfang Hospital, Southern Medical University and Dr. Lin Wu and Dr. Jian Bai of Berry Oncology. The researchers collected cell-free DNA (cfDNA) samples from a total of 3,234 individuals, including 2,250 patients with liver cirrhosis (LC), 508 with HCC, and 476 healthy controls (CTRL) from 13 hospitals in 11 provinces in China.

Scientists employed a state-of-the-art next-generation sequencing (NGS) technology to acquire genome-wide 5-hydroxymethylcytosine (5-hmc), nucleosome footprint (NF), 5 end motif and fragmentation profiles of cfDNAs from all enrolled individuals. Researchers then used a logistic regression method to construct a weighted diagnostic model based on the performance of these four features.

The novel diagnostic model, called HIFI, achieved a sensitivity of 95.42%, a specificity of 97.83%, and an AUC of 0.996 in the study, a higher accuracy in differentiating HCC from liver cirrhosis than existing diagnosis tests measuring AFP or PIVKA-II. The method was also able to detect signs of liver cancer with 93.9% accuracy among individuals that had tested negative using an AFP test, and with 90.9% accuracy among those that tested negative using a PIVKA-II test.

These results can be achieved regardless of a patient's age, HBV status, Child-Pugh score, BCLC stage, tumor size, or AFP status. Furthermore, the HIFI model can also be used to screen for other major cancers, such as lung cancer.

HCC, the most common form of liver cancer, is the second most deadly cancer worldwide. Patients with liver cirrhosis are at the highest risk of developing HCC, and diagnosis at an early stage contributes to an improved prognosis with the possibility of curative treatment.

"Due to the low accuracy of current diagnostic methods, new non-invasive strategies for early HCC diagnosis in cirrhotic patients are urgently needed," said authors of the study. "The HIFI method exhibited an excellent performance for early-stage HCC diagnosis compared to other non-invasive methods, and has huge potential of HCC detection for cirrhotic patients."

According to Berry Oncology CEO Jun Zhou, the result of this study is a milestone for Berry Oncology in tumor screening and early detection. As the forerunner in the field of genomic testing of cancers in China, this research aligns with Berry's strategic road map will accelerate Berry's advances in early screening of high-risk and high-incidence tumors and can also contribute to similar goals set out in China's 13th Five-Year Plan.

In addition to Berry Oncology, authors of the study also hail from the National Center for Liver Cancer, Shanghai Eastern Hepatobiliary Surgery Hospital in Shanghai, the Memorial Sloan Kettering Cancer Center in New York, Mengchao Hepatobiliary Hospital of Fujian Medical University in Fuzhou, the Second Hospital of Shandong University in Jinan, Ningbo Hwamei Hospital in Ningbo, Nanfang Hospital in Guangzhou and more.

About Berry Oncology

Berry Oncology, founded in 2017 as a member company of Berry Genomics, focuses on genetic testing of cancers. Driven by the mission of "Diagnose all cancers early, treat all patients precisely", we have established for cancer patients and high-risk population a complete system of genetic testing products and services, including tumor genetic susceptibility analysis, early screening, as well as as companion diagnostics, response monitoring and prognosis prediction of both targeted and immune therapies. For cancer early detection, we have launched a series of clinical research projects for early screening and early diagnosis, covering liver cancer, lung cancer, gynecologic cancer, and etc. Berry Oncology has three centers (manufacture, supercomputing, R&D) located in Fuzhou and Beijing. We serve and cooperate with more than 600 Class 3A hospitals across the country, and have provided valuable genetic testing services for hundreds of thousands of patients.

For more information please visit:http://en.berryoncology.com/

About Berry Genomics

Founded in May 2010, Berry Genomics is a leading company in clinical genomics and life science in China. Berry Genomics is dedicated to research, development and commercialization of genetic test technologies in clinical applications. Berry Genomics aims to assist accurate diagnosis of diseases throughout the full human life circle, and to improve human health.

As a company with strong R&D capability, Berry Genomics pioneered the first NGS-based genetic test, NIPT, in China back in 2010. The company currently provides NGS- based tests for many genetic diseases and cancers from preconception to adulthood. Berry Genomics is leading in the clinical study of early clinical detection of liver cancer in the world. Exploring the use of the third-generation sequencing technology in both clinical field and scientific study is ongoing.

Berry Genomics has around 1500 employees dedicating to developing products and providing services for over 4000 organizations and facilities home and abroad, including hospitals, research institutions, universities and corporations.

Berry Genomics has been listed on A-share market in China since 2017 under the stock code: 000710.

For more information please visit:http://www.berrygenomics.com/

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SOURCE Berry Oncology Corporation

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Berry Oncology Announces Novel Diagnostic Method for Liver Cancer Achieved Greater Accuracy and Sensitivity Compared to Existing Non-Invasive Methods...

Recommendation and review posted by Bethany Smith

According to the World Health Organization (WHO) and the American Cancer Society, cancer accounts for about 1 in 6 deaths worldwide - more than HIV/AIDS, tuberculosis, and malaria combined. The report states that by 2040, the global burden is expected to reach 27.5 million new cancer cases and 16.2 million cancer deaths. A WHO estimate in 2018 projected that 1 in 10 Indians will develop cancer during their lifetime and 1in 15 will die of the disease.

About 5-10% of all cancers are hereditary and occur due to inheritance of a genetic variation/mutation within families, especially in close family members. Examples of such hereditary cancer syndromes, also called familial cancer syndromes, are hereditary breast and ovarian cancer syndrome, Li-Fraumeni syndrome, Cowden syndrome, and Lynch syndrome.

Unlike localized (somatic) genetic variations that are acquired over the course of one's life, inherited or familial variations are transmitted from one generation to another and can thus predict the increased lifetime risk of developing cancer. The presence of such variations can also indicate the early onset of the disease.

From a comprehensive cancer treatment point of view, the knowledge of hereditary gene mutations has a plethora of applications including better disease management and application of preventive strategies such as risk-reducing surgeries, which can improve survival. Asymptomatic carriers can also be benefitted from chemoprevention and enhanced surveillance approaches.

There are about 100 genes that are associated with hereditary cancer development and information about mutations in these genes can be used to tailor prevention, surveillance, and treatment of cancer. Genetic testing to detect such inherited variations helps an Oncologist in making a well-informed treatment decision.

Hereditary cancers are known to have a poor-prognosis- meaning these cancers are comparatively more aggressive. This information can aid in better clinical management decisions. Further, mutations in certain genes increase response to drugs and thus contribute to precision/personalized medicine wherein patients are treated based on the mutations identified in them.

For example, women diagnosed with HBOC (Hereditary Breast and Ovarian Cancer syndrome), with a mutation in the BRCA1/2 gene, respond to targeted treatment using Poly ADP-ribose polymerase (PARP) inhibitors.

Considering the multiple advantages of identifying genetic variations in hereditary cancers, the health care fraternity globally, has been trying to make genetic testing more affordable and widely available to the public, enabling easier diagnosis, management, and treatment of this disease.

Sequencing of multiple cancer susceptibility genes simultaneously has now become available through multi-gene panel testing in a rapid and cost-effective manner. This is a breakthrough that will result in a new era of personalized healthcare for hereditary cancers in the decades to come.

-Article by Dr. Lakshmi Mahadevan, Principal Scientist, MedGenome Labs Ltd

READ | Advanced colon cancer: Why waiting may cost you your life

READ | World Cancer Day 2021: Theme, history and significance

READ | World Cancer Day 2021: Breast cancer scenario in India

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What genes tell us about the risk of developing cancer - India Today

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Pune, India, Feb. 22, 2021 (GLOBE NEWSWIRE) -- Gene Panel Market Overview

As per Market Research Future (MRFR), the Global Gene Panel Marketis expected to register aCAGR of12.16%to reachUSD4.34 Billion by 2025.

Increased cancer rates and genetic testing benefits will drive growth in the global market for genetic testing. Due to increased incidences of chronic illnesses and the increase in tailor-made gene panels for specific diseases, the market growth is exponential. In addition to market growth, a rise in initiatives across the world and DNA analysis benefits are anticipated to drive the gene panel market.

The thriving biotechnology industry coupled with increased awareness of advanced technologies is one of the main reasons the region has become a primary medical service provider. Although the gene panel testing is accurate, the test's limitations and inaccuracy are expected to impede the growth of the market. Furthermore, the stringent regulations and safety concerns regarding gene panels are likely to stifle this market in the immediate future. Regardless, ongoing research and development of gene sequencing are expected to lead to the rising need for genetic testing.

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COVID-19 Analysis:

The COVID-19 pandemic has heightened interest in the complex interaction between human host factors and pathogen factors. The spectrum of phenotypes associated with the SARS-CoV-2 infection, ranging from the absence of symptoms to severe systemic complications, raised the question of the extent to which the variable response to coronaviruses (CoVs) is influenced by the variability of the hosts' genetic background. Thus, the covid 19 pandemic has led to a rise in the research and development initiatives and investments towards genetics that could be helpful to design and conduct effective studies and, in turn, to find possible healthcare strategies aimed at facing the current COVID-19 pandemic, consequently creating new opportunities for market players of the global gene panel market.

Competitive Landscape:

The Prominent Players in the Global Gene Panel Market are:

The players operating in the global market have tried to innovate, develop, and acquire properties.

Browse In-depth Market Research Report (220 Pages) on Gene Panel: https://www.marketresearchfuture.com/reports/gene-panel-market-6100

Segmental Analysis:

The Global Gene Panel Market has been segmented by product & service, Technique, Design, Application, and End User.

Based on product & service, the market has been bifurcated into test kits and testing services.

The global gene panel market has been bifurcated into an amplicon-based approach and a hybridization-based approach based on technique.

The global gene panel market has been bifurcated into predesigned gene panels and customized gene panels based on design.

Based on application, the global gene panel market has been classified as cancer risk assessment, diagnosis of congenital disorders, and pharmacogenetics.

The global gene panel market based on end-user has been divided into hospitals, diagnostic laboratories, and others.

Regional Insights:

North America Leads with Established Sectors while APAC to Rise Substantially.

The Americas would be the largest market for gene testing at the end of the forecast period. The increasing prevalence of cancer and the growing presence of well-established players support the Americas' dominance in the coming years. North America dominated the global gene panel market due to its dominant position in cancer and rare diseases and the increase in applications for NGS-based and clinical research in the region. Such as many leading genomics researches and leading NGS providers in this region.

Europe created significant growth in the cancer medication market because of rising cancer rates. The rising incidence of cancer in people will, in part, drive the development of the cancer market. Moreover, the presence of a well-established healthcare sector and global market players leads to growth opportunities in Europe over the review period.

The Asia-Pacific region is projected to be the fastest-growing due to projected increases in life expectancy and the number of those who need healthcare. With increasing cancer prevalence and its resulting health care costs and the federal government's investment in cancer research and development, the market is likely to grow. Companies involved in genetic services can target India and China due to increased prevalence of infectious diseases.

Inability to fully advance in the biotechnology industry would have decreased business growth in the Middle East and Africa due to a lack of technological expertise. Due to the limited number of biotech consulting services available worldwide, the Middle East and Africa have the smallest global market share. Nevertheless, the gene panel market is expected to experience gradual growth in the Middle East and Africa due to the increased spending on healthcare and the growing number of people seeking genetic tests. The UAE, Saudi Arabia and Kuwait are expected to push the Middle East & African market. In comparison, the African region will see modest growth over the next decade.

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Industry Updates:

January 2021: Swift Biosciences released an S-gene panel for SARS-CoV-2 that covered the majority of viral genes even with very low levels of virus detection. The S gene controls the spike protein which allows the virus to attach to cells and affect transmissibility; mutations in the concerning variants have been discovered in the U.K., South Africa, Brazil, Denmark, and the United States. The panel will enable rapid scaling of the new strains' surveillance efforts by labs using the Illumina system and can be run by any lab using the Illumina system (NGS).

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Market Research Future (MRFR)is a global market research company that takes pride in its services, offering a complete and accurate analysis with regard to diverse markets and consumers worldwide. Market Research Future has the distinguished objective of providing the optimal quality research and granular research to clients. Our market research studies by products, services, technologies, applications, end users, and market players for global, regional, and country level market segments, enable our clients to see more, know more, and do more, which help answer your most important questions.

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Gene Panel Market Is Estimated to Reach USD 4.34 Billion by 2025 at CAGR of 12.16% | Market Research Future (MRFR) - GlobeNewswire

Recommendation and review posted by Bethany Smith

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimers disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

The protein, called brain-derived neurotrophic factor or BDNF, is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is particularly important in brain regions susceptible to degeneration in AD.

In previous published research, principal investigator Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, and colleagues described the prevention and reversal of brain cell degeneration and death in animal models.

Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimers disease the entorhinal cortex and hippocampus was able to reverse the loss of connections and to protect from ongoing cell degeneration, said Tuszynski. These benefits were observed in aged rats, aged monkeys and amyloid mice.

Amyloid mice are genetically engineered to inherit a mutation in the gene encoding the amyloid precursor protein, and as a result develop amyloid plaques aggregates of misfolded proteins in the brain that are considered a hallmark characteristic of AD.

BDNF is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

But BDNF is not easy to work with. It is a large molecule and cannot pass through the blood-brain barrier. As a result, researchers will use gene therapy in which a harmless adeno-associated virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt production of therapeutic BDNF in nearby cells.

The injections are precisely controlled to contain exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures.

The three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

This is the first safety and efficacy assessment of AAV2-BDNF in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called nerve growth factor (NGF) found heightened growth, axonal sprouting and activation of functional markers in the brains of participants.

The BDNF gene therapy trial in AD represents an advance over the earlier NGF trial, said Tuszynski. BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. In addition, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.

Despite billions of dollars of research investment and decades of effort, there are only two symptomatic treatments for AD. There is no cure or approved way to slow or stop progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Numerous clinical trials are ongoing to assess pharmaceutical remedies. Tuszynski said gene therapy, which debuted in 1980 and has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

We hope to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa), Tuszynski said.

BDNF gene therapy has the potential, unlike other AD therapies currently under development, to rebuild brain circuits, slow cell loss and stimulate cell function. We are looking forward to observing the effects of this new effort in patients with AD and MCI.

For more information on this Phase I clinical trial, contact Michelle Mendoza at 858-249-3015 or email alphastemcellclinic@ucsd.edu

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First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer's Disease - UC San Diego Health

Recommendation and review posted by Bethany Smith

WATERTOWN, Mass., and Research Triangle Park, N.C., Feb. 17, 2021 /PRNewswire/ --Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced the initiation of a Phase 1 dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.

"We are pleased to further evaluate ImmTOR's ability to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing by having initiated this dose-escalation study of SEL-399," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "This trial builds upon our strong preclinical data in non-human primates and marks the first time that ImmTOR in conjunction with an AAV capsid has been dosed in humans, which is a significant milestone. Data from this study will inform the design of future clinical trials in patients as we seek to unlock the full potential of gene therapy."

The dose-escalation trial of SEL-399 is designed to evaluate the safety and preliminary efficacyof ImmTOR in gene therapy. The study, being conducted in healthy volunteers at the SGS Life Sciences Clinical Pharmacology Unit in Antwerp, Belgium, plans to enroll up to 45 subjects to investigate increasing doses of ImmTOR and EMC-101. Subjects will be randomized in a 3:1 ratio of ImmTOR plus empty AAV8 capsid to empty capsid alone. Preliminary efficacy will be measured by assessing levels of AAV8-specific neutralizing antibodies.

Jude Samulski, Ph.D., chief scientific officer and co-founder of AskBio said, "By determining the dose at which ImmTOR is able to inhibit the formation of AAV-specific antibodies,this study could be a significant first step toward overcoming some of the unwanted immune responses associated with gene therapies. We look forward to using these findings to inform future studies as we work to develop strategies for repetitive dosing of AAV, thus extending durability of expression."

Selecta and AskBio expect to report initial results from this clinical trial in the fourth quarter of 2021.

AboutSelecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the body's natural self-tolerance in autoimmune diseases. The company's first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visitwww.selectabio.com.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020,is a fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more atwww.askbio.comor follow us onLinkedIn.

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of43.5 billion euros. Capital expenditures amounted to2.9 billion euros, R&D expenses to5.3 billion euros. For more information, visit http://www.bayer.com.

AskBio Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio's pipeline of development candidates; AskBio's collaboration with Selecta; AskBio's clinical trials, including its ability to enroll subjects, the timing of any such trials and any potential side effects; whether ImmTOR will be able to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing; the timing of and results from the SEL-399/101 trial; whether the SEL-399/101 study could be a significant first step in overcoming the immunogenicity concerns associated with gene therapies; AskBio's strategies for repetitive dosing of AAV, extending durability of expression; AskBio's goal of developing life-saving medicines aimed at curing genetic diseases; and the potential benefits of AskBio's development candidates to patients. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects ofSelecta Biosciences, Inc.("the company"), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy, the ability of the Company and AskBio to develop gene therapy products using ImmTOR and AskBio's technology, the novelty of treatment paradigms that the Company is able to develop, whether the observations made in non-human primate study subjects will translate to studies performed with human beings, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the company's plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the company's intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the company's ability to grow its strategic partnerships, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "hypothesize," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human primates, the unproven approach of the company's ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the company's product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the company's inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the company's recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the "Risk Factors" section of the company's most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with theSecurities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the company's views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

SOURCE Asklepios BioPharmaceutical, Inc.

http://www.askbio.com

Original post:
Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR in Gene Therapy - PRNewswire

Recommendation and review posted by Bethany Smith

The deal, which is coming fast on the heels of the CROs January 2021 acquisition of antibody discovery company, Distributed Bio, is expected to close in the first quarter of 2021.

Charles River said the acquisition will establish it as a scientific partner for cell and gene therapy (CGT) development, testing, and manufacturing providing clients with an integrated solution from basic research through CGMP production.

It noted the Memphis biotechs services, primarily cell therapy and plasmid production, serve a market worth about US$1.5bn globally.That market is expected to grow annually by at least 25% over the next five years, said the CRO.

Jim Foster, Charles Rivers CEO, commented."This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions."

In a Q4 2020 earnings call yesterday, Foster said the COVID-19 pandemic has also enhanced the global focus on scientific innovation, which is generating biomedical breakthroughs across multiple therapeutic areas, including for COVID-19 vaccines.

This innovation has fueled continued investment in and the proliferation of more complex research techniques involving advanced drug modalities, such as cell and gene therapies.

The complexity of these new modalities is increasing our clients' reliance on a high science outsourcing partner like Charles River. To enhance our ability to meet our clients' needs in these emerging areas of scientific innovation and to take advantage of the significant growth opportunities that these advanced drug modalities present, we are expanding our portfolio and scientific expertise through a combination of acquisitions, strategic partnerships and internal investments.

In November 2019, Charles River singled out CGT as the fastest growing component of its biologics business. The following month, it revealed a US$380m (340m) deal to buy CGT specialist, HemaCare, a company that had worked on three cell therapies approved in the US - Kymriah (tisagenlecleucel), Yescarta (axicabtagene ciloleucel) and Provenge (sipuleucel-T).

Then in August last year the CRO announced the acquisition of Memphis based Cellero, (formerly Key Biologics and Astarte Biologics), a provider of cellular products for cell therapy developers and manufacturers worldwide, for US$38m.

The Cellero deal, said Charles River, would complement the HemaCare business by boosting the supply of critical biomaterials, including a wide range of human-derived primary cell types to support the discovery, development, and manufacture of cell therapies.

See the rest here:
Charles River acquires Cognate BioServices to expand cell and gene therapy programs - BioPharma-Reporter.com

Recommendation and review posted by Bethany Smith

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimers disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

The protein, called brain-derived neurotrophic factor or BDNF, is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is particularly important in brain regions susceptible to degeneration in AD.

In previous published research, principal investigator Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, and colleagues described the prevention and reversal of brain cell degeneration and death in animal models.

Mark TuszynskiMark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimers disease the entorhinal cortex and hippocampus was able to reverse the loss of connections and to protect from ongoing cell degeneration, said Tuszynski. These benefits were observed in aged rats, aged monkeys and amyloid mice.

Amyloid mice are genetically engineered to inherit a mutation in the gene encoding the amyloid precursor protein, and as a result develop amyloid plaques aggregates of misfolded proteins in the brain that are considered a hallmark characteristic of AD.

BDNF is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

But BDNF is not easy to work with. It is a large molecule and cannot pass through the blood-brain barrier. As a result, researchers will use gene therapy in which a harmless adeno-associated virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt production of therapeutic BDNF in nearby cells.

The injections are precisely controlled to contain exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures.

The three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

This is the first safety and efficacy assessment of AAV2-BDNF in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called nerve growth factor (NGF) found heightened growth, axonal sprouting and activation of functional markers in the brains of participants.

The BDNF gene therapy trial in AD represents an advance over the earlier NGF trial, said Tuszynski. BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. In addition, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.

Despite billions of dollars of research investment and decades of effort, there are only two symptomatic treatments for AD. There is no cure or approved way to slow or stop progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Numerous clinical trials are ongoing to assess pharmaceutical remedies. Tuszynski said gene therapy, which debuted in 1980 and has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

We hope to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa), Tuszynski said.

BDNF gene therapy has the potential, unlike other AD therapies currently under development, to rebuild brain circuits, slow cell loss and stimulate cell function. We are looking forward to observing the effects of this new effort in patients with AD and MCI.

Read more here:
UC San Diego: First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimers Disease - India Education Diary

Recommendation and review posted by Bethany Smith

HACKENSACK, N.J., Feb. 18, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to endDuchenne muscular dystrophy (Duchenne), andDuchenne UK, a UK-based patient organization,are pleased to announce ProfessorKanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of their Joint Research Grant Call of 2020. The full title of the research project is "Targeting the innate immune system to block acute inflammatory and chronic immune response to transgene and AAV vector in DMD".Professor Nagaraju's research will receive funding from the organizations in the amount of $350,000.

These are promising times for research into Duchenne muscular dystrophy (Duchenne). Several companies are now testing an approach that uses a shortened dystrophin gene to replace the faulty dystrophin gene in Duchenne. This is known as gene transfer using micro-dystrophin, or more commonly, gene therapy. The companies are using viruses known as AAVs (adeno-associated viruses) to deliver the therapy.

However, challenges exist in getting this treatment to the entire Duchenne population. This is mainly because of immune responses: some patients have pre-existing antibodies to the AAVs. This means they will not, currently, be able to have the treatment because their bodies will recognize the virus and stop it from delivering the micro-dystrophin to the cells. In addition, as gene therapy is a new treatment, it is not yet clear if another dose will be required at a later stage, and it is not currently possible to re-dose with the same AAV.

This is why Duchenne UK & PPMD launched a call for projects last year that would specifically address this challenge.

The organizations received a large number of proposals, and three were taken forward for final review from a panel of highly qualified, specialized scientists. They looked at a wide variety of factors, including significance to the Duchenne community, and the ability to translate the research into treatments for patients.

Professor Nagaraju's research is looking at blocking the mechanism by which the body is able to recognise an AAV virus and mount an immune response to it. Importantly, he is using medicines that are already in use in humans, in an approach known as repurposing.

If this approach were successful, it would allow more micro-dystrophin to get to the cells, potentially requiring a lower dose of the AAV than is currently being administered in the trials. It may also allow patients who have already been dosed with gene therapy to receive further doses. Further to this, by using repurposed drugs, this treatment should be more easily transferable to patients. Professor Nagaraju believes that "targeting initial immune recognition pathways is one way to improve efficacy and safety profiles of AAV mediated gene therapy".

PPMD's Founding President & CEO, Pat Furlong, and Duchenne UK's CEO, Emily Crossley explained in a joint statement:"Supporting patients and accelerating innovative research is at the heart of what we do at Duchenne UK and PPMD. We are pleased to partner with each other and award this grant. Gene therapy is offering great promise, but there are challenges associated with the immune response which are limiting the rate of progress and a barrier to ensuring all patients can have access to these potentially transformative therapies.We would like to thank all those who participated and supported our Joint Grant Call and are very much looking forward to working with Professor Nagaraju on this vitally important project for the Duchenne community."

To learn more about PPMD's innovative research agenda and our investment portfolio, visit PPMD's website.

About Parent Project Muscular Dystrophy

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won four FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

About Duchenne UK

Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim to end Duchenne.

Duchenne UK are funding research that's focused on getting treatments to those affected now as well as pushing for an effective treatment in the future.

Duchenne UK connects leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. They connect families with each other to create a network of mutual support and to pool resources, knowledge and experience.

For more information about Duchenne UK: visit http://www.duchenneuk.org.

SOURCE Parent Project Muscular Dystrophy (PPMD)

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Duchenne UK and Parent Project Muscular Dystrophy Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne Muscular Dystrophy -...

Recommendation and review posted by Bethany Smith

The new gene therapy target,GJB2 coding for the Connexin 26 protein, has been added to Sensorions development portfolio: with the target the third candidate to emerge from the R&D collaboration with Institut Pasteur. It represents the largest gene therapy opportunity for the French biotech to date.

The GJB2 program will focus on major new markets with an estimated patient population of 300,000 children and adults in Europe and the US alone.

Sensorion, a French clinical-stage biotech based in Montpellier, was founded in 2009 to develop novel therapies to restore, treat and prevent hearing loss disorders.

The GJB2 program draws on new research from Institut Pasteur which shows that the same genes that underly congenital deafness are also involved in severe forms of presbycusis (age-related hearing loss). These forms of presbycusis appearing to be monogenic types of hearing loss that can be potentially treated by gene therapy.

Although the types ofGJB2mutations in children and adults may differ, Sensorion says gene therapy could potentially provide a solution to both.

Mutations inGJB2are believed to alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.

Institut Pasteur research now shows three pathologies related to GJB2 mutations: congenital deafness;age-related hearing loss in adults; and progressive forms of hearing loss in children. Sensorion will prioritize the latter two forms, saying it is the first company to address these needs and offering the potential of large market opportunities.

The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the "genetic landscape" of hearing loss," saidNawal Ouzren, CEO of Sensorion.

"It was clear that mutations in the GJB2 gene are important in severe to profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.

Sensorions collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications. Its USHER-CT gene therapy development program aims to restore inner ear function for patients suffering from Usher Syndrome Type 1 by providing a healthy copy of the USH1G gene coding for the SANS protein.

Meanwhile, the OTOF-GT gene therapy development program seeks to restore hearing in people with Otoferlin deficiency, one of the most common forms of congenital deafness.

Both of these have been proved in concept in preclinical studies.

Originally posted here:
Sensorion and Institut Pasteur announce new gene therapy collaboration - BioPharma-Reporter.com

Recommendation and review posted by Bethany Smith

Dublin, Feb. 15, 2021 (GLOBE NEWSWIRE) -- The "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Over time, several gene therapies have been developed for the treatment of both simple and complex genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma, ZyntegloT and Collategene) to date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods and genome editing and manipulation technologies, have enabled the development of novel therapy development tools/platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

The report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. GENE DELIVERY VECTORS

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

6. MARKET OVERVIEW

7. COMPETITIVE LANDSCAPE

8. MARKETED GENE THERAPIES

9. KEY COMMERCIALIZATION STRATEGIES

10. LATE STAGE GENE THERAPIES

11. EMERGING TECHNOLOGIES

12. KEY THERAPEUTICS AREAS

13. PATENT ANALYSIS

14. MERGERS AND ACQUISITIONS

15. FUNDING AND INVESTMENT ANALYSIS

16. CLINICAL TRIAL ANALYSIS

17. COST PRICE ANALYSIS

18. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

19. DEMAND ANALYSIS

20. MARKET FORECAST AND OPPORTUNITY ANALYSIS

21. VECTOR MANUFACTURING

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN

23. CONCLUSION

A Selection of Companies Mentioned Include:

For more information about this report visit https://www.researchandmarkets.com/r/c6r4ih

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Gene Therapy Market Outlook to 2030 - by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of...

Recommendation and review posted by Bethany Smith

WILMINGTON, Mass.--(BUSINESS WIRE)--Charles River Laboratories International, Inc. (NYSE: CRL) announced today that it has signed a definitive agreement to acquire Cognate BioServices, Inc., a premier, cell and gene therapy contract development and manufacturing organization (CDMO), for approximately $875 million in cash, subject to customary closing adjustments. The transaction is expected to close by the end of the first quarter of 2021, subject to regulatory requirements and customary closing conditions.

James C. Foster, Chairman, President and Chief Executive Officer of Charles River Laboratories, commented, Cognate BioServices presents a unique opportunity to expand into a high-growth, value-added sector of the CDMO market and enhance our ability to meet our clients needs in emerging areas of scientific innovation. This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions and enabling us to achieve our goal of establishing a single scientific partner to provide biopharmaceutical clients with an integrated solution to help accelerate their cell and gene therapy programs from discovery and non-clinical development through commercialization.

Because of the synergistic fit with Charles River, the market growth potential, and the emerging role of advanced drug modalities as treatments for oncology and rare disease, we believe Cognate will meaningfully enhance our long-term revenue and earnings growth potential. We look forward to welcoming Cognates dedicated employees to the Charles River family, Mr. Foster concluded.

Cognate is a premier, cell and gene therapy CDMO offering comprehensive manufacturing solutions for cell therapies, as well as for production of plasmid DNA and other inputs in the CDMO value chain. The planned acquisition will establish Charles River as a premier scientific partner for cell and gene therapy development, testing, and manufacturing, providing clients with an integrated solution from basic research and discovery through CGMP production. Cognate has extensive experience producing various cell types and technologies used in cellular immunotherapy and immuno-oncology, regenerative medicine, and advanced cell therapy. Headquartered in Memphis, Tennessee, Cognate has operations in North America and Europe with over 500 employees.

Strategic Rationale

The acquisition of Cognate will expand Charles Rivers scientific capabilities into the emerging, high-growth cell and gene therapy CDMO sector, establishing a comprehensive solution from discovery and non-clinical development through CGMP manufacturing in advanced drug modalities.

Cognate will be particularly synergistic with the Companys Biologics Testing Solutions business. It will enable clients to seamlessly conduct analytical testing, process development, and manufacturing for advanced modalities with the same scientific partner, enabling them to achieve their goal of driving greater efficiency. Clients will also have access to the Companys cellular products as the starting point for their cell therapy programs and will be able to work with Charles River through every step of the research and early-stage development process before moving into CGMP production with Cognate, accelerating clients speed to market for advanced drug modalities.

Additional Financial and Transaction Details

Based on the anticipated completion of the acquisition by the end of the first quarter, Cognate is expected to add approximately $110 million to Charles Rivers 2021 consolidated revenue for the partial year. The transaction is expected to be neutral to non-GAAP earnings per share in 2021, and accretive thereafter. Items excluded from non-GAAP earnings per share are expected to include all acquisition-related costs, which primarily include amortization of intangible assets, advisory fees, certain costs associated with efficiency initiatives, and certain third-party integration costs.

The acquisition and associated fees are expected to be financed through Charles Rivers existing credit facility and cash. The Company is evaluating further optimizing its debt structure which could be used to finance the acquisition and for general corporate purposes.

Cognate is expected to be reported as part of Charles Rivers Manufacturing Support segment.

Advisors and Cognate OwnershipGordon Dyal & Co., LLC is acting as the exclusive financial advisor to Charles River. Davis Polk & Wardwell LLP is acting as Charles Rivers transactional legal counsel, and Weil, Gotshal & Manges LLP is acting as antitrust counsel. Dark Horse Consulting Group, Inc. is acting as Charles Rivers strategic advisor.

Cognate is supported by its majority shareholder, EW Healthcare Partners, as well as minority shareholders, Medivate Partners, BlackRock, and a sovereign wealth fund. Morgan Stanley & Co. LLC is acting as the exclusive financial advisor and Kirkland & Ellis LLP is acting as legal counsel to Cognate and its shareholders.

Use of Non-GAAP Financial Measures

This news release contains non-GAAP financial measures, such as non-GAAP earnings per diluted share, which exclude the amortization of intangible assets, integration costs, advisory fees, and other charges related to our acquisitions and expenses associated with evaluating acquisitions. We exclude these items from the non-GAAP financial measures because they are outside our normal operations. There are limitations in using non-GAAP financial measures, as they are not prepared in accordance with generally accepted accounting principles, and may be different than non-GAAP financial measures used by other companies. In particular, we believe that the inclusion of supplementary non-GAAP financial measures in this news release helps investors to gain a meaningful understanding of our core operating results and future prospects without the effect of these often-one-time charges, and is consistent with how management measures and forecasts the Company's performance, especially when comparing such results to prior periods or forecasts. We believe that the financial impact of our acquisitions (and in certain cases, the evaluation of such acquisitions, whether or not ultimately consummated) is often large relative to our overall financial performance, which can adversely affect the comparability of our results on a period-to-period basis. In addition, certain activities, such as business acquisitions, happen irregularly and the underlying costs associated with such activities do not recur on a consistent basis. Non-GAAP results also allow investors to compare the Companys operations against the financial results of other companies in the industry who similarly provide non-GAAP results. The non-GAAP financial measures included in this news release are not meant to be considered superior to or a substitute for results of operations prepared in accordance with GAAP. The Company intends to continue to periodically assess the potential value of reporting non-GAAP results consistent with applicable rules and regulations. A reconciliation of the effect of this transaction on non-GAAP earnings per share for 2021 to the most directly comparable GAAP financial measure has not been included because it is impracticable to determine the allocation of the purchase price for the proposed acquisition and other necessary adjustments at this time.

Caution Concerning Forward-Looking Statements

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words such as anticipate, believe, expect, will, may, estimate, plan, outlook, and project and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements include statements in this news release regarding the acquisition of Cognate BioServices, expectations regarding the timing of the closing of the acquisition, and Charles Rivers expectations with respect to the impact of Cognate on the Company, its product and service offerings, client perception, revenue, revenue growth rates, and earnings per share; Charles Rivers projected future performance including revenue and earnings per share; as well as Charles Rivers future growth in the area of cell and gene therapy CDMO services. Forward-looking statements are based on Charles Rivers current expectations and beliefs, and involve a number of risks and uncertainties that are difficult to predict and that could cause actual results to differ materially from those stated or implied by the forward-looking statements. Those risks and uncertainties include, but are not limited to, the ability to successfully complete the acquisition of Cognate, our ability to successfully integrate Cognate, and risks and uncertainties associated with Cognates products and services which are in areas in which Charles River does not currently operate. A further description of these risks, uncertainties, and other matters can be found in the Risk Factors detailed in Charles River's Annual Report on Form 10-K as filed on February 11, 2020 and the Quarterly Report on Form 10-Q as filed on October 29, 2020, as well as other filings we make with the Securities and Exchange Commission. Because forward-looking statements involve risks and uncertainties, actual results and events may differ materially from results and events currently expected by Charles River, and Charles River assumes no obligation and expressly disclaims any duty to update information contained in this news release except as required by law.

About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit http://www.criver.com.

Continue reading here:
Charles River Laboratories to Acquire Cognate BioServices to Create a Premier Scientific Partner for Cell and Gene Therapy Development - Business Wire

Recommendation and review posted by Bethany Smith

-- Gene editing expert Charles Albright, Ph.D., joins as chief scientific officer --

-- Gene therapy development expert Petra Kaufmann, M.D.,joins as chief medical officer

WALTHAM, Mass., Feb. 17, 2021 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and non-rare diseases, today announced the completion of its leadership team. Collectively, the executives represent a diverse team of proven leaders in gene therapy who have successfully developed novel platforms and translated them to medicines that have made a transformative difference in the lives of those affected by devastating diseases.

Our calling is to broaden the reach of gene therapies for patients in need, and this starts with our proven leadership team. Members of our team have been at the forefront of the field and come from a variety of eminent organizations to innovate in the gene therapy science and programs at Affinia Therapeutics, said Rick Modi, chief executive officer at Affinia Therapeutics. We believe in a vastly different future medical era of one-time potentially curative medicines for patients affected by diseases, rare and non-rare. But realizing this vision requires moving beyond conventional AAV serotypes. At Affinia Therapeutics, we are uniquely positioned to engineer novel vectors and gene therapies that direct tissue tropism and have the potential to improve clinical efficacy, as well as immunogenicity and safety. Together, Dr. Albright, Dr. Kaufmann and the rest of our leadership team will leverage their extensive experience to advance our platform and programs.

Dr. Albright joins Affinia Therapeutics from Editas Medicine, where he served as executive vice president and chief scientific officer and led the development of the CRISPR gene editing technology platform. In this role, he industrialized and matured the platform and translated it to pioneering programs through Investigational New Drug (IND) submissions that led to clinical trials. Prior to joining Editas, Dr. Albright spent over 12 years at Bristol Myers Squibb, where he most recently held the position of vice president, genetically defined diseases and genomics. As a research leader at both biotech and large pharmaceutical companies, he has honed his expertise across a range of therapeutic areas including neurology, neuromuscular, cardiovascular, ophthalmology and oncology. Dr. Albright earned a Ph.D. in biology at MIT and was a postdoctoral fellow at the Whitehead Institute.

Dr. Kaufmann is an expert in translational medicine and clinical development focused on neuroscience, rare disease and gene therapy. She recently served as senior vice president and head of clinical development, analytics and translational medicine at Novartis Gene Therapies. She directed clinical development activities that included the global development of Zolgensma and translational strategy for several pipeline programs. This followed many years of clinical research and development positions in academia and at the National Institutes of Health (NIH), where she held leadership roles of increasing responsibility, most recently as director of the Office of Rare Diseases Research. Dr. Kaufmann earned an M.D. at the University of Bonn and an M.S. in biostatistics at Columbia University, where she also trained in neurology and served as a tenured faculty member, advancing research and caring for patients.

It is exciting to see the platform generate advances beyond the conventional AAV serotypes discovered many years ago, said Dr. Albright. Affinia Therapeutics platform for engineering next-generation vectors with specific pharmacodynamic properties enables us to explore the potentially curative benefits of gene therapy in new indications. I look forward to leading the scientific team as we advance these insights into translational opportunities in the clinic and, ultimately, into transformative therapies for patients.

I am thrilled to join this team of gene therapy experts who are helping to broaden the reach of gene therapies, said Dr. Kaufmann. I look forward to putting my clinical research and development experience into practice as we translate our differentiated AAV vectors into transformative medicines for people suffering from diseases that have been inadequately addressed by conventional AAVs and traditional therapies.

Dr. Albright and Dr. Kaufmann join the following individuals on Affinia Therapeutics leadership and scientific advisory team:

This team has collectively authored more than 450 publications, holds more than 15 patents, held meaningful roles on more than 15 BLAs, supplemental BLAs and product launches, and has executed on more than $16 billion in value for IPOs and M&As.

About Affinia TherapeuticsAt Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating rare and non-rare diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies with potentially improved tissue tropism, cell specificity, immunogenicity and safety. With our innovative science, we are working to broaden the reach of life-changing gene therapies to meaningful numbers of patients with an initial focus on central nervous system (CNS) and muscle diseases with significant unmet need. http://www.affiniatx.com.

Affinia Therapeutics Contacts

Investors: investors@affiniatx.com

Media: media@affiniatx.com

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Affinia Therapeutics Announces Addition of Gene Therapy Scientific and Medical Experts to Leadership Team to Advance Novel Gene Therapy Platform and...

Recommendation and review posted by Bethany Smith

Igor Golovniov/SOPA Images/LightRocket via Getty Images

Charles River Laboratories will acquire Cognate BioServices, a cell and gene therapy contract development and manufacturing organization, in an $875 million cash deal that will significantly expand the companys capabilities in the high-growth cell and gene therapy sector.

In addition to expanding Charles Rivers capabilities in the cell and gene therapy space, the company said the acquisition of Memphis, Tenn.-based Cognate will also establish a comprehensive solution from discovery and non-clinical development through CGMP manufacturing in advanced drug modalities. Cognate has a global presence with more than 500 employees. In January, Cognate announced plans to significantly expand cell and gene therapy manufacturing capacity, laboratory space, warehousing capabilities, and increase office support at its facilities inthe United StatesandEurope.

The company has extensive experience producing various cell types and technologies used in cellular immunotherapy and immuno-oncology, regenerative medicine and advanced cell therapy. Its primary area of expertise is in CGMP cell therapy manufacturing, which processes a variety of cellular products and other starting materials to develop and produce allogeneic (donor-derived) and autologous (patient-derived) cell therapies. Cognate also produces plasmid DNA, which is a foundational tool used in the development of gene-modified cell therapies and gene therapies, as well as other CDMO inputs, Charles River said.

James C. Foster, president and chief executive officer of Charles River Labs, said Cognate provides a synergistic fit for Charles River. He said bringing Cognate under the companys umbrella presents a unique opportunity to expand the companys capabilities and also enhance its offerings to clients in emerging areas of scientific innovation.

Additionally, Charles River said the addition of Cognate will complement its existing Biologics Testing Solutions business. Cognates capabilities will enable clients to seamlessly conduct analytical testing, process development, and manufacturing for advanced modalities with the same scientific partner, enabling them to achieve their goal of driving greater efficiency, the company said.

The addition of Cognate is also expected to provide a significant financial boost to Charles River. Cognate is expected to generate annual revenue of approximately $140 million in 2021, and is expected to grow at least 25% annually over the next five years, the company said.

This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions and enabling us to achieve our goal of establishing a single scientific partner to provide biopharmaceutical clients with an integrated solution to help accelerate their cell and gene therapy programs from discovery and non-clinical development through commercialization, Foster said in a statement. Because of the synergistic fit with Charles River, the market growth potential, and the emerging role of advanced drug modalities as treatments for oncology and rare disease, we believe Cognate will meaningfully enhance our long-term revenue and earnings growth potential.

The acquisition is expected to move quickly. Charles River anticipates the closing of the deal by the end of the first quarter of 2021. Based on the anticipated completion of the acquisition by the end of the first quarter, Cognate is expected to add approximately $110 million to Charles Rivers 2021 consolidated revenue for the partial year.

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Charles River Dives Deep Into Cell and Gene Therapy With Cognate BioServices Acquisition - BioSpace

Recommendation and review posted by Bethany Smith

A new Market Research Report by Facts and Factors Market Research (fnfresearch.com), on Cancer Gene Therapy Market Overview By Trends, Industry Top Manufactures, Size, Industry Growth Analysis & Forecast Till 2026 added to the flourishing data archive is in place to provide readers with innate detailing on market developments, comprising a detailed market overview, vendor landscape, market dimensions, vendor landscape as well as in-depth SWOT and PESTEL assessment, besides other internationally approved market assessment guidelines that play crucial roles in growth dissemination.

The report Cancer Gene Therapy Market 2020-2026 focuses on outlining and narrating the key influencing elements for the growth of the market. It also proposes a thorough study of the market stature (revenue), market share, key market segments, distinct geographic regions, dominant market players, and prime industry trends. The aim of this report is to portray the forthcoming market trends and revenue prognosis for the global Cancer Gene Therapy market for the next five years.

According to the research report, Global Cancer gene therapy market is expected to grow at a CAGR of 23% and is anticipated to reach around USD 3,400 million by 2026. Gene therapy is a method for curing or avoiding the substitution of mutated genes of health diseases. This replacement enables cells to produce valuable proteins.

We Have Recent Updates of Cancer Gene Therapy Market in Sample Copy @ https://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

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Cancer Gene Therapy Market by Top Manufacturers (2020-2026)

Vigene Biosciences

Sirion Biotech

Bluebird bio

Cellectis

Ziopharm

Cobra

Uniqure

Finvector

Sarepta Therapeutics

To Get an Additional List of Key Market Players Included in this Report, Request a Free PDF Brochure: https://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

The Market Player Analysis based on some of the below Factors:

Pre & Post Impact of COVID-19 Pandemic on Cancer Gene Therapy Market

The COVID-19 outbreak has affected economies and industries in various countries due to lockdowns, travel bans, and business shutdowns. The decrease in the overall market growth due to COVID-19 is also affecting the Post growth of the Cancer Gene Therapy market owing to the shutting down of factories, obstacles in the supply chain, and a downturn in the world economy.

This report presents a comprehensive overview, market shares, and growth opportunities of Cancer Gene Therapy market by product type, application, key manufacturers, and key regions and countries. In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges, and the risks faced by key manufacturers and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

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Points Covered in the Report

The points that are discussed within the report are the major market players that are involved in the market such as market players, raw material suppliers, equipment suppliers, end users, traders, distributors and etc.

The complete profile of the companies is mentioned. And the capacity, production, price, revenue, cost, gross, gross margin, sales volume, sales revenue, consumption, growth rate, import, export, supply, future strategies, and the technological developments that they are making are also included within the report. This report analyzed 12 years of data history and forecast.

The growth factors of the market are discussed in detail wherein the different end users of the market are explained in detail.

Data and information by market player, by region, by type, by application, etc., and custom research can be added according to specific requirements.

The report contains the SWOT analysis of the market. Finally, the report contains the conclusion part where the opinions of the industrial experts are included.

The market research report also offers information on potential investment opportunities, strategic growth market analysis, and probable threats that will adhere to the client to systematically and creatively plan out the business models and strategies. The critical data analysis in the Cancer Gene Therapy market report is laid out in an upright way. This means that the information is represented in form of infographics, statistics, and uncomplicated graphs to make it an effortless and time-saving task for the client.

(**The consulting and implementation services segment to account for the highest market share during the forecast period.**)

The scope of the Cancer Gene Therapy Market Size report has a wide spectrum extending from market scenarios to comparative pricing between major players, cost, and profit of the specified market regions. The numerical data is supported by statistical tools such as SWOT analysis, BCG matrix, SCOT analysis, and PESTLE analysis. The statistics are depicted in a graphical format for a clear picture of facts and figures.

The report provides a complete view of the Cancer Gene Therapy market and encompasses a detailed type of portfolio and strategic developments of key vendors. To know the competitive landscape of the Cancer Gene Therapy market, an analysis of Porters five forces model is done. The study cover market attractiveness analysis, in which type, source type, and application segments are specialized based on the market size, growth rate, and attractiveness.

The report study further includes an in-depth analysis of industry players market shares and provides an overview of leading players market position in the Cancer Gene Therapy sector. Key strategic developments in the Cancer Gene Therapy market competitive landscape such as acquisitions & mergers, inaugurations of different products and services, partnerships & joint ventures, MoU agreements, VC & funding activities, R&D activities, and geographic expansion among other noteworthy activities by key players of the Cancer Gene Therapy market are appropriately highlighted in the report.

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Table of Contents

Research Coverage:

The Cancer Gene Therapy market has been segmented based on offering, technology, end-use application, and end-user. It also provides a detailed view of the market across four main regions: North America, Europe, APAC, and RoW.

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Most Elevated Read 2020 on Cancer Gene Therapy Market To Witness Robust Expansion Throughout The Forecast Period 2026 KSU | The Sentinel Newspaper -...

Recommendation and review posted by Bethany Smith

Basel, February 17, 2021 Novartis today announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD). The project brings together Novartis drug discovery and gene therapy expertise with the Gates Foundations charitable objectives to expand access to healthcare in low-resource settings in an effort to address this potentially life-threatening genetic disease.

Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease, said Jay Bradner, a hematologist and President of the Novartis Institutes for BioMedical Research (NIBR). This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.

SCD is a hereditary blood diseaseone of the oldest known and most common genetic disorders. The disease affects millions around the world, with over 300,000 born with the condition annually1,2. It disproportionately affects those of African descent, and sub-Saharan Africa bears roughly 80 percent of the disease burden3. It also is common among people with ancestry from South America, Central America, and India, as well as several Mediterranean countries, such as Italy and Turkey.

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 disease1,4,5,6. The disease can lead to acute episodes of pain known as sickle cell pain crises, or vaso-occlusive crises, as well as life-threatening complications7,8,9. The resulting, frequent hospitalizations associated with sickle cell disease combined with an overall lack of specialized care places a significant burden on patients and their families, healthcare systems, and the overall economy. Even with todays best available care, SCD continues to drive premature deaths and disability.

While the genetic cause of SCD has been known for decades, its only recently that the medical world has gained the tools to potentially fix the errant gene that causes the disease. Current clinical-stage gene-based therapies require extracting cells from a patient, altering those cells in a laboratory, and then reintroducing them to the patient through a complex procedure akin to a bone marrow transplant. The lab and manufacturing facilities and hospital infrastructure for such procedures often do not exist in the areas where SCD is most prevalent, excluding the vast majority of those with the disease from these life-changing gene therapies.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, said Trevor Mundel, President of Global Health at the Gates Foundation. Whats exciting about this project is that it brings ambitious science to bear on that challenge. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.

Novartis envisions developing an accessible in vivo gene therapy for SCD that could potentially be administered once, directly to the patient, without the need to modify the cells in a lab. This would have the advantage of mitigating the need for long or repeated hospital stays or specialized lab infrastructure. To facilitate the research, the Gates Foundation has agreed to provide funding support for a research team within NIBR wholly dedicated to developing an approach to delivering this potential treatment. Novartis will provide in-kind support and access to its suite of technologies and resources.

In addition to research funding, the Gates Foundation lends its long history and experience in global health to this collaboration. As part of the early drug design strategy, Novartis will prioritize addressing access and distribution hurdles posed by limited healthcare infrastructure in low- and middle-income countries and the funding agreement includes specific provisions to support global access to any resulting innovations.

Novartis is proud to lead this effort to find an accessible genetic therapy for sickle cell disease, with support from the Bill & Melinda Gates Foundation, said Lutz Hegemann, Group Head of Corporate Affairs & Global Health for Novartis. In keeping with our purpose, we firmly believe we can use science and innovation to reimagine the way SCD is treated for patients around the world.

The agreement builds on the Novartis commitment to improve the lives of patients with sickle cell disease through the development of new treatments, including crizanlizumab, strategic partnerships with government and non-government organizations, and support for such interventions as newborn screenings and the distribution of existing drugs.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, potentially, can, will, could, would, believe, commitment, pipeline, to discover, aims, to address, promise, envisions, to facilitate, to provide, lead, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, including crizanlizumab, or regarding the collaboration to discover and develop an accessible in vivo gene therapy for sickle cell disease. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release, including crizanlizumab, will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that the activities and efforts described in this release will be achieved or succeed, in the expected time frame, or at all. In particular, our expectations regarding such products and efforts could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com/.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact media.relations@novartis.com

References

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Novartis Media RelationsE-mail: media.relations@novartis.com

Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

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Novartis and the Bill & Melinda Gates Foundation collaborate to discover and develop an accessible in vivo gene therapy for sickle cell disease -...

Recommendation and review posted by Bethany Smith

Newark, NJ, Feb. 17, 2021 (GLOBE NEWSWIRE) -- As per the report published by Fior Markets, theglobal genome editing market is expected to grow from USD 5.37 billion in 2020 to USD 17.36 billion by 2028, at a CAGR of 15.8% during the forecast period 2021-2028.

Some of the factors that drive the growth of the global genome editing market are expanding genetic research funding in North America, rising synthetic gene demand in North America & Asia Pacific regions, growing R&D expenditure for biotechnology, technological developments in gene editing, presence of strong pipeline products, increasing application fields of genomics, and rising production of GM crops. New products are being developed to assist in improving DNA targeting, with better flexibility, higher specificity, and enhanced AAV packaging.

With the outbreak of a global pandemic, many have faced salary cuts and job losses. Covid-19 has become a threat to the entire world's economy and has negatively impacted all kinds of markets, including the genome editing market. With less disposable income and broken supply chains, the market's growth is expected to slow down. A shortage of skilled professionals due to medical workers being shifted to covid-19 frontlines also negatively impacts the market. Other market growth hindering factors are low public awareness in underdeveloped nations and strict government regulations.

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Key players operating in the global genome editing market include Thermo Fisher Scientific, Horizon Discovery Limited, GenScript, Eurofins Scientific, Editas Medicine, CRISPR Therapeutics, Oxford Genetics, Synthego, Vigene Biosciences, Integrated DNA Technologies, OriGene Technologies, Transposagen Biopharmaceuticals, Agilent Technologies, Genecopoeia, and Precision Biosciences, among others. To gain a significant market share in the global genome editing market, the key players are now focusing on adopting strategies such as product innovations, mergers & acquisitions, recent developments, joint ventures, collaborations, and partnerships. Thermo Fisher Scientific and Horizon Discovery Limited are some of the biggest players in the global genome editing market.

CRISPR/Cas9 segment dominated the market and held the largest market share of 32.8% in the year 2020

The technology segment consists of ZFN, TALENs/MegaTALs, CRISPR/Cas9, and others. The CRISPR/Cas9 segment dominated the market and held the largest market share of 32.8% in the year 2020. This is primarily due to CRISPR's better ease of use as compared to TALEN & ZFN, and patient-specific treatment characteristics for illnesses like cystic fibrosis.

Ex-vivo segment dominated the market and held the largest market share of 55.3% in the year 2020

Based on delivery method, the global market has been divided into in-vivo and ex-vivo. Ex-vivo segment dominated the market and held the largest market share of 55.3% in the year 2020. The large share of this segment is attributed to key factors such as control ease in DNA modification, and precise regulation of nuclease expression for highly accurate gene editing.

Cell line engineering segment dominated the market and held the largest market share of 34.1% in the year 2020

The application segment includes cell line engineering, plant genetic engineering, animal genetic engineering, and other applications. Cell line engineering segment dominated the market and held the largest market share of 34.1% in the year 2020. Some of the vital factors that drive market growth in this segment are rising government as well as private funding, increasing global awareness, and expanding industry attention on stem cell research.

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Biotechnology & pharmaceutical companies segment dominated the market and held the largest market share of 45.3% in the year 2020

Based on end-use, the genome editing market has been segmented into contract research organizations, academic & government research institutes, and biotechnology & pharmaceutical companies. Biotechnology & pharmaceutical companies segment dominated the market and held the largest market share of 45.3% in the year 2020. The large share of this segment can be attributed to factors like rising prevalence of cancer & other diseases, and the increasing number of gene engineering research projects being carried out for advancement in novel therapeutics.

In-house segment dominated the market and held the largest market share of 60.9% in the year 2020

Based on service, the market has been divided into in-house and contract. The in-house segment dominated the market and held the largest market share of 60.9% in the year 2020. Large companies like Sigma Aldrich Corporation and Thermo Fisher engineer cell lines in-house due to the availability of financial resources, technology, and facilities.

Regional Segment Analysis of the Genome Editing Market

On the basis of geography, the global genome editing market has been classified into North America, Europe, South America, Asia Pacific, and Middle East and Africa. The North America region accounted for the major market share of 48.2% in the year 2020 and is anticipated to grow throughout the projected period. The large share of this segment is attributed to key factors such as R&D activities in gene therapy, increasing cases of cancer & other diseases, increasing funding & research grants, and rising usage of GM (genetically modified) crops in the region. On the other hand, the Asia-Pacific region is expected to grow at the highest CAGR during the forecast period 2021-2028. This growth is owing to factors like advancing medical science and rising disposable income.

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The global genome editing market is analyzed on the basis of value (USD Billion). All the segments have been analyzed on a global, regional, and country basis. The study includes the analysis of more than 30 countries for each segment. The report offers an in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insight of the market. The study includes porter's five forces model, attractiveness analysis, raw material analysis, and competitor position grid analysis.

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Global Genome Editing Market Is Expected to Reach USD 17.36 Billion by 2028 : Fior Markets - GlobeNewswire

Recommendation and review posted by Bethany Smith

ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --

REGENXBIO Inc. (Nasdaq: RGNX) reported at the Angiogenesis, Exudation, and Degeneration 2021 conference additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up (LTFU) study. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.

"The continued durability of treatment effect up to three years after RGX-314 administration highlights the potential of RGX-314 as a one-time treatment option for patients with wet AMD. The results from the Phase I/IIa trial of RGX-314 using subretinal delivery have informed the key design elements of our pivotal program, in which we plan to conduct two randomized, well-controlled clinical trials, enrolling approximately 700 patients total," said Steve Pakola, M.D., Chief Medical Officer ofREGENXBIO.

"I am excited about this data out to three years, which demonstrates that one-time treatment with RGX-314 has the potential to result in long-term stability to improvement of visual acuity outcomes and retinal anatomy, while alleviating treatment burden," said Allen C. Ho, M.D., Director of Retina Research at Wills Eye Hospital and Mid Atlantic Retina and investigator surgeon in the RGX-314 clinical trials. "In our practice, and as reported by multiple real-world studies, we see many patients losing vision due to lack of compliance with standard of care, which requires frequent anti-VEGF injections. I look forward to further evaluating the effects of RGX-314 in ATMOSPHERETM, the first pivotal trial of a gene therapy for the treatment of wet AMD."

Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD Using Subretinal Delivery

In the Phase I/IIa trial of RGX-314, 42 patients with severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated across all cohorts, with 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5. The most common nonserious adverse events in the eye were generally assessed as mild (87%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3 through 5, retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. Retinal hemorrhage was observed in 26% of patients and is an anticipated event in patients with severe wet AMD. There have been no reports of clinically-determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Summary of Data for Cohorts 4 and 5

Today's update includes data from Cohorts 4 and 5 as of January 22, 2021. Each cohort enrolled 12 patients each at doses of 1.6x1011 GC/eye and 2.5x1011 GC/eye, respectively.

Patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314 demonstrated stable visual acuity with a mean Best Corrected Visual Acuity (BCVA) change of +1 letters and -1 letters from baseline, respectively, as well as decreased central retinal thickness (CRT), with a mean change of -46 m and -93 m, respectively.

There was a meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the mean annualized injection rate during the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.4 injections over 1.5 years following administration of RGX-314, a 58.3% reduction in anti-VEGF treatment burden. Patients in Cohort 5 received a mean of 1.7 injections over 1.5 years following administration of RGX-314, a reduction in anti-VEGF treatment burden of 81.2%.

In Cohort 4, four out of 12 (33%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of +2 letters at 1.5 years. Eight out of 11 (73%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of -2 letters at 1.5 years.

Summary of Long-Term Follow-Up (LTFU) Study Data

Following the Phase I/IIa trial, patients are encouraged to enroll in a LTFU study to assess safety and efficacy up to five years after RGX-314 administration. Patients in the LTFU study have scheduled visits every six months for the first year and then annual visits until the end of the study. Patient management is per physician discretion. Data collected during the scheduled study visits include safety, BCVA, and CRT. In addition, chart reviews are conducted at each scheduled study visit to collect the number of retina specialist visits and anti-VEGF injections each patient has received since the prior scheduled study visit.

As of January 22, 2021, RGX-314 continued to be generally well-tolerated in patients enrolled in the LTFU study, with no new drug-related ocular adverse events reported.

All six patients from Cohort 3 of the Phase I/IIa trial enrolled in the LTFU study, and long-term treatment effect was demonstrated over three years. These patients demonstrated a mean BCVA improvement of +12 letters from baseline at three years. Retinal anatomy as measured by machine-read CRT remained stable at three years compared to the two-year timepoint.

Patients also demonstrated long-term reductions in anti-VEGF treatment burden over three years with a mean annualized rate of 2.4 anti-VEGF injections after administration of RGX-314, which is a reduction of 66.7% from the mean annualized injection rate during the 12 months prior to administration of RGX-314. Three out of six (50%) patients received no anti-VEGF injections over three years following one-time administration of RGX-314. Four out of six (67%) patients have received no anti-VEGF injections from nine months to three years after RGX-314 administration. The four patients who did not receive anti-VEGF injections after nine months demonstrated a mean BCVA improvement from baseline of +11 letters at three years.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About the Phase I/IIa Clinical Trial of RGX-314 and Long-Term Follow-Up Study

RGX-314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, dose-escalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SDOCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect. After completion of the Phase I/IIa clinical trial, patients are encouraged to enter a Long-Term Follow-Up study to continue to follow safety and efficacy for a total of 5 years following administration of RGX-314.

About ATMOSPHERE

ATMOSPHERE is a multi-center, randomized, active-controlled trial to evaluate the efficacy and safety of a single-administration of RGX-314 versus standard of care in patients with wet AMD. The trial is designed to enroll 300 patients at a 1:1:1 ratio across two RGX-314 dose arms (6.4x1010 genome copies (GC)/eye and 1.3x1011 GC/eye delivered subretinally) and an active control arm of monthly intravitreal injections of ranibizumab (0.5 mg/eye). The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks. Secondary endpoints of the trial include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections. Patient selection criteria will include patients with wet AMD who are responsive to anti-VEGF treatment and will be independent of preexisting neutralizing antibody status. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The trial will be conducted at approximately 60 clinical sites based in the United States, with over 100 retinal surgeons.

AboutREGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to accurately predict how long REGENXBIO's existing cash resources will be sufficient to fund its anticipated operating expenses, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at http://www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

SCS Microinjector is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Contacts: Tricia TruehartInvestor Relations and Corporate Communications347-926-7709ttruehart@regenxbio.com

Investors:Brendan Burns, 212-600-1902brendan@argotpartners.com

Media:David Rosen, 212-600-1902david.rosen@argotpartners.com

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SOURCE REGENXBIO Inc.

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REGENXBIO Announces Additional Positive Interim Phase I/IIa and Long-Term Follow-Up Data of RGX-314 for the Treatment of Wet AMD - BioSpace

Recommendation and review posted by Bethany Smith

Dublin, Feb. 17, 2021 (GLOBE NEWSWIRE) -- The "I-O Therapy Combinations Spurring Growth of the Global Immuno-oncology Market" report has been added to ResearchAndMarkets.com's offering.

This study explores the global immuno-oncology (I-O) market's evolution from 2020 to 2025. An extensive analysis is provided on the growth opportunities envisioned by the analyst to help stakeholders leverage their momentum and disrupt the market by offering best-in-class solutions.

The study considers oncology agents for which the immune system is the basis of their actions, including monoclonal antibodies, cell and gene therapies, bioengineered vaccines, recombinant products, small molecules, and oncolytic viruses. Classified into 5 major therapy types - immunomodulators, adoptive cell therapy, cancer vaccine, oncolytic virus, and antibody-based targeted therapies - the analysis provides revenue forecasts by the global market and product category. The agents are classified into various therapy types based on the mechanism of action (MOA). Concerning pipeline products, we have considered industry-sponsored products under development.

The oncology therapeutic segment is expected to witness minimal impact from the COVID-19 pandemic because of the criticality of treating cancer patients and the segment's role as a major revenue-generator for pharmaceutical companies. The analyst have considered the impact of COVID-19 and two scenarios for the sales forecast: original scenario and COVID-19 impact scenario.

In the original scenario, the I-O market is expected to reach $132.66 billion by 2025 growing at a rate of 11.6% and witness minimal impact from the COVID-19 pandemic, with 2020 accounting for the majority of the decline. After 2022, the market will positively recuperate and experience a slight increase in revenue due to rebounding R&D budgets and accelerated developments of late-stage clinical candidates.

Research Highlights

Major Themes Covered in This Study:

Key Topics Covered:

1. Strategic Imperatives

2. Executive Summary

3. Market Overview-Immuno-oncology

4. Immuno-oncology Value Chain and R&D Expenditure-Immuno-oncology

5. Clinical Trial Overview-Immuno-oncology

6. Regulatory and Reimbursement Scenario-Immuno-oncology

7. Key Marketed and Pipeline Products Dashboard-Immuno-oncology

8. Growth Opportunity Analysis-Global Immuno-oncology Market

9. Growth Opportunity Analysis-Immunomodulators

10. Growth Opportunity Analysis-Adoptive Cell Therapy

11. Growth Opportunity Analysis-Antibody-based Targeted Therapies

12. Growth Opportunity Analysis-Cancer Vaccine and Oncolytic Virus

13. Growth Opportunity Universe-Immuno-oncology

14. Key Company Profiles-Immuno-oncology

15. Next Steps

For more information about this report visit https://www.researchandmarkets.com/r/x1ktob

Read more here:
IO Therapy Combinations Spurring Growth of the Global Immuno-oncology Market, 2020 Report - Upcoming Product Launches in Adoptive Cell Therapy Segment...

Recommendation and review posted by Bethany Smith

Provided by Washington Examiner

President Biden ushered in his presidency alongside Americas first female vice president, pledging a message of unity and womens advancement. Yet, with a simple pen stroke, Biden set women back and effectively crippled the rights and dreams of millions of women and girls.

How? By signing an executive order to allow biological males to compete in female athletics. By siding with the few, Biden is decimating womens rights and their futures.

He need not look further than what this policy has already done to girls at the state level, and he should reverse course on this injustice rather than bringing it to the federal level.

Simply put, the policy he champions hurts Americas girls. Female athletes in Connecticut have been forced to face this sad reality for years. Just ask Selina Soule and Alanna Smith. Soule and Smith are two student athletes who, like many, dreamed of achieving success in sports. In 2017, after years of grueling practices and long nights, their goals finally seemed within reach.

Then the finish line was moved in the name of equity.

When these women took their spots at the starting line, they were lined up next to two biological males who identify as females. No matter their identity, the facts remain: Their genetics and physical bodies are equipped with athletic abilities unachievable to women. Unsurprisingly, these males defeated Soule and Smith and their dreams in the process.

This all began when the Connecticut Interscholastic Athletic Conference started accepting boys who identify as female in its competitions. The results have predictably been disastrous for the state of female sports.

These male athletes have gone on to claim 15 womens track championship titles in two years. And theyre not alone. Currently, 16 states allow full transgender participation in high school athletics, while an additional 10 states require medical documentation to participate.

In these female competitions, who won 15 titles? The boys.

Who set 13 individual track meet records? The boys.

Who lost out on their goals and dreams? The girls.

Fast forward to 2021, and our new administration is dead set on taking this injustice to the next level. Bidens nationwide inclusion mandate seeks to damage the very rights his party claims to champion.

This has gone on long enough.

As someone who enjoyed playing in sports in high school and coaching high school track for many years, I am well aware of the damage this executive order places on current and future generations of women. Sadly, this action by Biden will water down our hard-fought rights and protections while effectively denying female athletes fair competition.

When we misconstrue Title IX rules and morph them into an alternate reality, our girls are the ones who end up losing. This is unacceptable.

For our society to grow stronger, we need to build up the traits that make each of us unique, not walk back decades of female victories in order to minimize our differences. Americas girls deserve better than to become victims of political correctness.

Biden said, I will be a president for all Americans. If thats the case, he should stop working to reverse the rights of half of them.

Rep. Vicky Hartzler represents Missouri's 4th Congressional District in the U.S. House of Representatives.

Tags: Opinion, Op-Eds, Transgender Issues, Sports, Gender Issues, Joe Biden, Capitol Views

Original Author: Rep. Vicky Hartzler

Original Location: President Biden, stop working to reverse the rights of half of America

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President Biden, stop working to reverse the rights of half of America - msnNOW

Recommendation and review posted by Bethany Smith

The gut microbiota is the community of bacteria, fungi, viruses, and other microorganisms that make their home in the human digestive tract.

In recent years, scientists have discovered an astonishing range of interactions between the gut microbiota and human health. These include effects on mood, eating disorders, and immunity.

A recent study reported by Medical News Today also found associations between particular bacterial species and the risk of developing diabetes, heart disease, and obesity.

Bacteria in the gut produce an enzyme called bile salt hydrolase (BSH) that may be essential to many of their beneficial effects.

For example, scientists have linked high BSH activity to decreased inflammation, reduced blood cholesterol levels, and protection against colon cancer and urinary tract infections.

Scientists still know surprisingly little about the complex interactions between the diet, the gut microbiota, and BSH activity.

Recently, an international team of researchers set out to study the effects of prebiotics insoluble dietary fibers that promote the growth of friendly bacteria on the activity of BSH.

They also wanted to develop a clinical test for assessing the enzymes activity in people with inflammatory bowel disease.

Microbiologists currently use indirect techniques to gauge the enzymes activity, for example by analyzing fecal samples or culturing bacteria in the lab. But these efforts do not reflect the diversity of bacteria and the chemical complexity of their natural environment, which varies according to where they live in the gastrointestinal tract.

So the team, led by researchers at the University of Missouri, in Columbia, and the Swiss Federal Institute of Technology, in Lausanne, developed a noninvasive chemical probe that measures the activity of BSH along the entire length of the gut.

They have published their findings in the journal Science Advances.

The probe relies on a natural compound called luciferin, which emits light in the presence of oxygen, and the enzyme luciferase, which originates in fireflies.

To create the probe, the researchers chemically caged the luciferin by combining it with a small molecule that shields it from luciferase.

They designed the cage so that only the enzyme BSH can liberate the luciferin molecule from the probe. As a result, the amount of light that a fecal sample produces in the presence of luciferase is proportional to the amount of BSH along the length of the gut.

The scientists first tested their probe on samples in the lab. A female volunteer then swallowed two capsules containing the probe, and these successfully measured the enzymes activity as they passed through her gut.

Until now, we have not had any ways to noninvasively monitor activity in the intact gastrointestinal tract, given the unique chemical environment, variable distribution, and highly dynamic nature of the gut microbiota, says Dr. Elena Goun, an associate professor in the universitys department of chemistry and the senior author of the paper.

In another experiment, in mice, the team used the probe to test the ability of different types of prebiotic supplement inulin and fructo-oligosaccharides (FOSs) to boost the production of BSH.

They discovered that inulin produced no significant change in BSH levels in the animals guts, but FOSs doubled these levels.

Dr. Goun explains the significance of her teams results:

Prebiotics are often used in combination with probiotics to enhance their functions in the body. [] We show, for the first time, that certain types of prebiotics alone are capable of increasing [BSH] activity of the gut microbiota, which, among other health benefits, has been shown to decrease inflammation, reduce blood cholesterol levels, and protect against colon cancer and urinary tract infections.

The senior researcher adds that there could also be important cost benefits, as prebiotics are less expensive to produce and store than probiotics.

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Noninvasive probe monitors health of 'friendly' gut bacteria - Medical News Today

Recommendation and review posted by Bethany Smith

Columnist Walter Suza encourages to strengthen diversity and inclusion in institutions by beginning at an individual level.

There will be progress. Like after the Obama administrations effort to make college more affordable and accessible to low-income students and students of color.

There will be setbacks. Like after Trumps executive order threatened diversity training in U.S. colleges.

The impact of such external factors is hard to miss. Yet its internal factors that can escape our attention because theyre deeply ingrained in our normal way of operating.

Imagine a stellar high school student with a dream of attending her favorite university, which is predominantly white. The aspiring student visits the university and hears more about various great programs and activities. The visit seals the deal.

The student enrolls as an undeclared major.

A class in philosophy introduces the student to George Berkeley, who tried to put God in the center of philosophy, and Immanuel Kant, who believed that moral principles must be followed unconditionally.

Martin Heidegger also captures the students attention with his idea that: We do not experience mere sensations abstracted from the real objects of the world; rather, our experiences are of everyday objects in all their richness and complexity.

From Heidegger's thinking, the student recognizes that theres complexity in our humanness, and richness in our cultures, backgrounds and experiences.

Yet upon looking around the department, the student sees more vividly that the department is homogeneously white. The student wonders: How can I experience the richness and complexity of a professor of color if I dont interact with such a professor?

The student looks white and also is Jewish, and upon further research she learns that Heidegger was supportive of the Nazis. The student wonders: Why was this not mentioned in class?

Unfortunately, what was also left out in the discussion was the fact that modern philosophy has ties to racism and bigotry. Upon this realization, the student decides to take a course in genetics.

In genetics she learns about the amazing work of James Watson and Francis Crick to unravel the structure of the genetic code. The student is elated that knowledge of the genetic code made it possible to create the COVID-19 mRNA vaccine.

Upon further research the student finds out that Watson and Crick relied on the work of a female scientist, Rosalind Franklin, yet Franklin wasnt considered for the Nobel Prize. Another student whos Black shares that Watson was stripped of his numerous accolades because of his racist views against Black people. The students wonder: Why were these things not acknowledged in class?

In the genetics class, the students read research papers on bone development and biological networks and are upset to discover that the terms master and slave are used with impunity in biology and computer science.

The Black student raises more concern about the term breeding used in crop and animal science. The term breeding reminds the students of a dark past in America involving the creation of slave breeding farms to supply Black slaves to plantations. The students wonder: Why is the term breeding still used to describe improvement of the genetics of animals and crop plants?

The students browse the internet and learn that in French, Spanish and Portuguese languages, the term breeding is not used, instead, the term improvement of plants is used.

The students meet at a coffee shop with other concerned students to discuss the use of offensive terms in courses. They also discuss circulation of hate speech on social media.

A student from journalism warns the group that controversial social media posts are protected by the First Amendment. The Black and Jewish students are appalled by this and ask: How about the protection of those affected by hate speech?

A professor in agronomy who happens to be at the coffee shop overhears the conversation and approaches the students and says:

I am sorry for the hurtful experiences you have endured in our institution. I have also been complicit in this. I was educated and Im working in the area we call plant breeding. I also use the term "breeding" in my teaching and research. Now I see better and I want to do something about this. I am sorry for perpetuating the pain.

The Black student turns to the professor and says: Imagine having a guest in our home. If we are good hosts, we would prepare a room for our guest, we would make sure our guest has food to eat, we would spend time with our guest, we would show that were interested in their well-being, we would make sure our guest feels safe in our home.

There is a lot of work to be done in academic institutions such as this one to make the learning environment safe.

The work starts at the individual level.

First by imagining being in the underrepresented and having to deal with racism. This will help us become aware of beliefs and stereotypes about those different from us.

Next, by looking inward for our own unconscious biases because learning that we have unconscious bias can help us to develop compassion.

Compassion helps us gain the courage to accept that prejudice lives and breathes in academia.

Compassion helps us become willing to address offensive content in our lectures and speeches.

Compassion helps us become willing to address controversial monuments on our campus.

Compassion helps us realize that we are at our best when we treat others the way we would like them to treat us.

Walter Suza is an adjunct associate professor in the department of agronomy.

Original post:
Suza: Self-reflection strengthens diversity and inclusion | Opinion | iowastatedaily.com - Iowa State Daily

Recommendation and review posted by Bethany Smith

While youre receiving treatment for cancer, some of the drugs you take can cause painful sores to develop inside your mouth. You can also get them if youve had a bone marrow (stem cell) transplant as part of your cancer care.

Although they often heal on their own, these mouth sores can make it uncomfortable to eat and talk. Well discuss what you can do to relieve the pain and prevent them from getting worse.

Mouth sores can be a common side effect of cancer treatment. The condition, known as stomatitis or mucositis, is an inflammation of the tissues inside your mouth.

Whitish, ulcer-like sores can form on your cheeks, gums, lips, tongue, or on the roof or floor of your mouth. Even if you dont develop mouth ulcers, you may have patches that feel inflamed and painful, as if theyve been burned.

Anyone who is receiving chemotherapy, radiation therapy, or a bone marrow (stem cell) transplant can develop mouth sores as a side effect of these treatments.

If you have dry mouth or gum disease, or if your teeth and gums are not well taken care of, you may be at a higher risk of getting mouth sores during your treatment. Women and people who smoke or drink alcohol are also at a higher risk, according to the Oral Cancer Foundation.

If youre receiving chemotherapy, the sores could begin forming anywhere from 5 days to 2 weeks after your treatment. Depending on the specific cause, the sores could go away on their own in a few weeks, or they could last longer.

Its important to find ways to manage your pain and to watch for signs of an infection. Cancer-related mouth sores can lead to weight loss, dehydration, and other serious complications.

Cancer cells can grow very quickly. The aim of cancer treatment is to stop or slow down that growth. The cells in the mucous membranes lining your mouth are also fast-growing cells, so cancer treatments affect them, too.

Cancer treatments also keep the cells in your mouth from being able to repair themselves efficiently when theyre damaged.

Radiation therapy can also damage the glands in your mouth that make saliva. A dry mouth is more susceptible to infections that cause mouth sores.

Chemotherapy and radiation can both change the microbiome in your mouth, upsetting the balance between good and bad bacteria. The growth of harmful bacteria in your mouth can also lead to mouth sores.

Sometimes cancer treatments suppress your immune system, which may make it more likely that youll get a bacterial, viral, or fungal infection that causes mouth sores. An older infection (such as the herpes simplex virus) can also suddenly flare up again.

If youve had a bone marrow (stem cell) transplant, sores may be a sign that youve developed a condition known as graft-versus-host disease (GVHD).

When this happens, the cells in your body are attacking the transplanted cells as though they were an unhealthy invader. According to research published in Journal of Clinical and Experimental Dentistry, short-term (acute) GVHD occurs in 50 to 70 percent of stem cell transplant cases and longer-term (chronic) GVHD is seen in 30 to 50 percent of cases.

The form of GVHD that causes mouth sores is usually mild, and doctors often treat it with corticosteroid medications.

Its important to talk with your doctor if you develop mouth sores after a stem cell transplant, as some kinds of GVHD can turn serious if left untreated.

There is a good chance that youll experience mouth sores at some point during your cancer treatment. Researchers estimate that 20 to 40 percent of those who have chemotherapy and 80 percent of those who have high-dose chemotherapy will develop mucositis afterward.

Still, there are steps you and your cancer care team can take to lower your risk, reduce the severity of the sores, and promote faster healing.

About a month before your cancer treatment begins, schedule an appointment with your dentist to make sure your teeth and gums are healthy. If you have cavities, broken teeth, or gum disease, its important to come up with a dental treatment plan to take care of these conditions so they dont lead to infections later, when your immune system may be vulnerable.

If you wear braces or dentures, ask your dentist to check the fit and remove any part of the device you dont need during your treatment.

Its very important to maintain good oral hygiene practices throughout your treatment to lower your risk of infection. Brush and floss gently but regularly, avoiding any painful areas. You can also ask your dentist whether a mouth rinse with fluoride is advisable in your case.

For certain kinds of chemotherapy (bolus 5fluorouracil chemotherapy and some high-dose therapies), your healthcare team may give you ice chips to chew for 30 minutes before your treatment. This type of cold therapy can lower your risk of getting mouth sores later.

During treatment of some blood cancers, doctors may give you injections of palifermin, also known as human keratinocyte growth factor-1 (KGF-1), to prevent mouth sores.

If youre scheduled to receive high-dose chemotherapy or radiotherapy, your cancer care team may prepare your mouth using low-level laser therapy beforehand to keep you from getting mouth sores.

For people who have radiation therapy for head and neck cancers, doctors may prescribe this medicated mouthwash to minimize mouth sores.

The length of time your mouth sores may last depends on the specific cancer treatment youve had. Here are some estimates broken down by treatment:

You may notice symptoms anywhere between a few days and a few weeks after your cancer treatment. Heres what you may see and feel as mucositis develops:

You may notice that the sores become slightly crusty as they heal. Its important to keep track of your symptoms and let your oncologist know if the sores arent healing on their own.

Contact your doctor right away if you:

Untreated mouth sores can lead to malnutrition, dehydration, and life-threatening infections.

There are a few different ways that you can help mouth sores heal and avoid prolonger pain or an infection.

While the sores are healing, its very important to keep the inside of your mouth clean to prevent an infection from developing.

The National Cancer Institute recommends that you gently clean your teeth every 4 hours and just before you go to sleep at night. Here are a few tips to consider:

If the pain from mouth sores is interfering with your ability to eat and drink, your doctor may treat the condition with a opioid mouthwash or one containing doxepin or lidocaine.

To ease discomfort and keep your mouth from feeling dry, you may want to try rinsing with a mild saltwater or baking soda solution. Heres how to make each of them:

Your cancer care team may recommend that you use a lubricating liquid (artificial saliva) to moisten the inside of your mouth if dryness is a problem. These liquids are usually gel-like. They coat your mouth with a thin film to help ease discomfort and promote healing.

Some people have found it useful to rinse with a blend of medications called the magic mouthwash. Formulas for this mouthwash vary, but most of them include a combination of medications to treat different symptoms, including:

Magic or miracle mouthwash solutions usually have to be prescribed by a doctor and prepared by a pharmacist, although some people mix up an over-the-counter version at home.

There isnt enough research to say for sure whether magic mouthwash works. If you think youd like to try it, talk with your oncologist or a healthcare professional about whether its a good idea for you.

Here are a few more things you can try at home that may help ease pain from mouth sores:

Mouth sores are one of the most common side effects of cancer treatment. Shortly after chemotherapy, radiation, or transplant treatments, painful, ulcer-like sores can form on the inside of your mouth.

These sores may go away on their own. If they dont, its important to seek medical treatment for them because they can lead to very serious complications.

Before you start cancer treatments, visit a dentist to make sure your teeth and gums are healthy. Keeping up good dental hygiene practices during and after cancer treatment will help limit mouth sores.

If the sores are keeping you from eating and drinking, talk with your oncologist about medications could relieve the pain and speed up the healing process, so you can enjoy a better quality of life during treatment.

Its really important to keep track of any sores in your mouth so you can reach out to your healthcare team if they dont improve. Sores that deepen or worsen can lead to serious even life-threatening complications.

More:
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SASKATOON -- An effort to increase stem cell donors within Black communities across Canada is being driven by a group of women whove had difficulty finding full genetic matches themselves.

Genetic matches are crucial for patients in need of stem cell transplants, such as those with leukemia and lymphoma, and matches are more commonly found within their own racial, ethnic and ancestral groups.

But the new Black Donors Save Lives campaign notes that fewer than two per cent of those in the Canadian Blood Services stem cell donor registry are Black.

And that decreases their chance of finding a match, campaign lead Sylvia Okonofua told CTVNews.ca in a phone interview. It becomes a numbers game for Black people on the stem cell waiting list, where its like finding a needle in a hay stack for them.

The recent University of Regina biochemistry graduate, with sights on becoming a hematologist, timed the virtual campaign to kick off during Black History Month.

It was overall frustrating to know that a patient from my community is so much less likely than other patients to be helped, she told CTVNews.ca. When you see that your people have a really, really low chance of being helped out, it takes you aback.

Okonofua noted part of the campaign uses TikToks, shareable infographics, and even an original song to get the message out and reach a wide audience.

And she said part of the outreach involves having Black stem cell recipients talk about their experiences with the health-care system and speak to the historical mistrust the Black community has towards the medical community.

She founded her campus chapter of Stem Cell Club, a non-profit organization with chapters across Canada which recruits Canadians as potential stem cell donors.

Registration for Black Donors Save Lives can be done online, where participants between the ages of 17 to 35 can fill out a questionnaire and have a swab kit mailed to their address. After they swab the inside of their cheeks and send the sample back, if there is a person in need, 90 per cent of donors will be asked to donate stem cells very similar to the way a person would be giving blood.

But a big difference is the donor is given a growth hormone a week before donation in order to increase the number of stem cells, as well as the process taking four to six hours.

Alternatively, one out of 10 donors will be asked if theyd like to donate stem cells via bone marrow surgery, which can take place over a day.

In 2017, Reve Agyepong experienced firsthand the lack of Black stem cell donors, to treat her sickle cell disease, which involve red blood cells becoming misshapen, which can block blood vessels and lead to damage to bones, brain, kidneys, and lungs, and can ultimately be fatal.

But Agyepong, who was born in Edmonton to Ghanaian parents, was fortunate to receive a stem cell transplant from her sister.

It is such a blessing to have a match within your own family because the percentages are just so low, she told CTVNews.ca by email. I am so fortunate to have found a match in my family or else transplant would have been off the table for me.

In fact, only one in four patients who need a stem cell transplant are able to find a matched donor within their family, with Black patients being less than half as likely as white patients to find a unrelated person they match with on a donor registry, according to the campaign.

For Jamaican-Canadian Dorothy Vernon-Brown, who helped inspire this months campaign, the current efforts are deeply personal. In 2013, she was diagnosed with acute myeloid leukaemia and was heartbroken to discover there were no stem cell matches in Canada's registry or internationally.

She ultimately received stem cells from her sister, who was a half-match, and has been spreading information to Black Canadians ever since, through her own advocacy group, Donor Drive for Dorothy.

Stem cell transplantation is a miracle for patients, and I wish people knew how easy it is to be a stem donor, she recounted on a Twitter thread for another stem cell awareness campaign. You could give someone an opportunity like my sister gave me, to be around and live the life I want. People want to live, so if that gift is in your hands, I appeal to you to see it as something significant to do in your life.

Okonofua and Vernon-Browns efforts are being aided by Dr. Warren Fingrut, a hematologist whos the director of the aforementioned Stem Cell Club.

He told CTVNews.ca in an email hes seen firsthand far too many patients from ethnic and racial minority groups in situations where they dont have fully-matched donors and are forced to seek other treatments.

I find this heart wrenching and I am very motivated to work to address this, Fingrut said.

That led to him founding his non-profit a decade ago, which has gone on to recruit more than 20,000 Canadians as stem cell donors, with more than 55 per cent being non-white. But in cases such as Vernon-Brown and others, those figures need to be much higher.

We started running national campaigns last year, focused on the recruitment of diverse peoples as donors, as well as males who are also preferred by transplant physicians (all else being equal) as they are associated with better outcomes for patients, Fingrut explained.

The campaign is also being done in partnership with several other groups, including the Katelyn Bedard Bone Marrow Association, Black Physicians of Canada, Black Medical Students Association of Canada and the National Black Law Students Association of Canada.

This campaign is one example of an initiative in the health-care sector, which seeks to address racial disparity impacting the care of Black patients, he wrote, noting Black people face many such disparities in access to care, and we want to see others in the health-care sector working with Black Canadians to tackle these issues and address them, in collaboration with Black communities.

Okonofua hopes next Black History Month, theyll be able to have in-person swabbing events in places of worship, community hubs, and cultural gatherings to show how easy it is.

Fingrut said this the first time his group has specifically engaged with one racial group and hopes to expand it to other ethnic and racial communities including South Asians, Indigenous peoples, and those of mixed ancestry in the near future.

Read the rest here:
Meet the women hoping to recruit more stem cells donors from Black communities - CTV News

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