On March 8, 2022, David Bennett died, two months after a team at the University of Maryland Baltimore transplanted a pig heart into his body. Such xenotransplantation has long occupied the public imagination, with one of the earliest examples being the myth of Icarus where the father-son duo grafted bird wings onto their bodies. Bennetts operation has helped reify the groundbreaking potential of xenotransplantation: how an unlimited supply of animal organs could render the transplant list, where 17 people die every day waiting for a donor, obsolete. This promise is enticingly attractive, but what is being lost in all the hype and triumphalism it has elicited?

For one, theres much we still dont know about xenotransplantation, according to Dr. Richard Pierson, Director of the Center for Transplantation Sciences at Massachusetts General Hospital. The Virginia-based company Revivicor used the CRISPR-Cas9 system to add in six genes and knock out four from the pig used for Bennetts operation, but we dont know precisely what the right pig is that we ought to be using, Pierson said. Perhaps the right one is the 3-gene pig from the German team, or maybe its a hypothetical pig with manifold other genetic modifications. To manage the threat of transplant rejection, doctors say theyll need to do more genetic tinkering with the pig heart, as well as more tinkering with the immunosuppressive regimen undergone by the patient. What is the safest way to immunosuppress someone whos had a xenotransplant? Pierson said. We dont know. The transplant protocol for Bennett was probably good enough, but nobody knows if it was the best.

Theres also the open question of whether xenotransplantation might enable zoonoses, or the jumping of pathogens across the species divide. Revivicor took extensive steps to ensure a clean pig, from performing a cesarean section of the pregnant sow, to not allowing the piglets to suckle their mother, to maintaining a hermetically sealed pig-in-the-bubble environment. But, despite all this external control, there are porcine endogenous retroviruses that remain and might be a problem according to Pierson. Hes quick to clarify that the existing evidence suggests these endogenous retroviruses dont get into actual humans in any productive way. We dont think thats going to be a major barrier, but it remains unknown, Pierson said.

These are all known unknowns, but Piersons main unanswered question is the unknown. Is there some pathogen out there? How will we know its there? How will we respond if we see it? he asked rhetorically. Revivicor can do all the testing it wants to ensure a clean pig, but how can they test for something they dont even know to look for? Theres going to be a tremendous amount to learn before all these questions are answered, Pierson said. But this does not mean we should abandon ship. For Pierson, it just means we should take cautious steps forward.

Bennetts operation inevitably draws historical comparisons to that of Baby Fae, a case that many agree was catastrophically premature. Baby Fae was a 12-day-old infant who, diagnosed with hypoplastic left heart syndrome, received a walnut-sized baboon heart at Loma Linda University in 1984. Baby Fae died 21 days after the operation because of transplant rejection, and her story became an ethical lightning rod, eliciting accusations of inadequate informed consent, medical hubris, and human experimentation. Of course, animal rights activists also raised the issue of exploiting nonhuman primates. Dr. Leonard Bailey, the surgeon behind the operation, dismissed these concerns, saying, When it gets down to a human living or dying, there shouldnt be any question.

But maybe there should be. These animals are very close to us. Theyre experiencing pain, friendship, and love. How can you experiment on them and use them for parts? asked Dr. Sharon Kaufman, Chair of the Department of Anthropology, History, and Social Medicine at the University of California San Francisco. Its the central question that has haunted animal experimentation since its inception: How are animals similar enough to be our model organisms yet different enough to be exploited for our ends?

According to Kaufman, xenotransplantation is an expression of our manifest destiny to expand the limits of human life beyond what was previously imaginable. Its the tyranny of potential whereby progress can march triumphantly, unimpeded by calls for reflection and pause. There used to be no potential. Now its all potential, Kaufman said. Thats whats changed in medicine over the past 50 to 60 years. If something is available, no matter how experimental, no matter how far-fetched, no matter if it even works, it becomes ethically appropriate, necessary, and standard.

Norman Shumway, the father of heart transplantation, once said xenotransplantation is the future of transplantation, and always will be. But, with science fiction now becoming our reality, we have to confront renewed questions about our relationship with animals and how we should navigate the interface between our species and others. As Pierson makes it clear, xenotransplantation isnt quite ready for showtime, but its speeding down to realize its potential, whether we like it or not. By cutting past the hype and triumphalism though, animal rights activists, anthropologists, and the public at large can help shape the path xenotransplantation takes, paving a way that is more informed, cautious, and as humane as possible.

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Inside the Pig Heart Transplant and Ethical Dilemma That Followed - Sentient Media

NEW YORK & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apertura Gene Therapy, a biotechnology company opening opportunities for treating debilitating diseases with limited options for patients, today announced that it has launched with a Series A financing of up to $67M from Deerfield Management Company to develop genetic medicines using platform technologies that address key limitations of genetic medicine delivery and expression. Deerfield Management has also committed additional operational support to further strengthen the companys ability to advance gene therapy discoveries.

Apertura is founded on a pair of platform technologies developed in the labs of Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, and Michael Greenberg, Ph.D., the Nathan Marsh Pusey Professor of Neurobiology and Chair of the Department of Neurobiology at Harvard Medical School (HMS). The companys platform leverages machine learning and high-throughput assays to engineer novel capsids, gene regulatory elements, such as promoters and enhancers, and payloads to simultaneously enhance multiple functions of gene therapies for greater translational potential.

With these platform technologies from the Broad Institute and Harvard University, Deerfield saw an opportunity to bring together and support a unique and comprehensive platform that could address technical challenges that have prevented gene therapy from reaching its full potential, said Dave Greenwald, Ph.D., Acting Chief Executive Officer of Apertura and Vice President, Business Development at Deerfield Management Company. While next-generation approaches to gene therapy have largely focused on the innovation of delivery vectors, Apertura has the potential to innovate simultaneously across delivery, expression, and payloads.

Ben Deverman, Scientific Founder of Apertura, said: When developing a gene therapy, it has been common to use naturally occurring serotype AAV capsids. The technology we have developed uses proprietary assays and machine learning to design custom AAV capsids that have the chosen characteristics for treating specific diseases, and we believe this approach will result in new and effective gene therapies.

Apertura has certain exclusive rights to AAV capsids developed in the Deverman Lab at the Broad Institute.

A separate sponsored research and licensing agreement with Harvard University, spearheaded by the Harvard Office of Technology Development, grants Apertura exclusive access to certain powerful methods of identifying cell type-specific genetic regulatory elements (GREs), including access to the Paralleled Enhancer Single-Cell Assay (PESCA) platform, developed in the Greenberg Lab at Harvard University.

A major challenge in developing effective gene therapies is having the payload of the therapy expressed at the correct level in target cells, said Greenberg. "The technology we have developed at Harvard Medical School overcomes this hurdle by targeting transgene expression to specific cell types, fine-tuning expression levels in these cells, and, at the same time, avoiding expression of the transgene in non-target cell types. The Greenberg labs PESCA platform was advanced to commercial readiness through the strategic support of the Q-FASTR program at HMS and the Blavatnik Biomedical Accelerator at Harvard University.

The two technology platforms have unique capabilities to simultaneously engineer AAV capsids to exhibit enhanced cellular tropism, evasion of pre-existing immunity, while maintaining and potentially improving manufacturability. The companys GRE platform focuses on GREs and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels. These capabilities together are expected to enable Apertura to develop best-in-class gene therapies designed for specific indications.

Our platform has the potential to unlock many new indications for gene therapy, said Kristina Wang, Director of Corporate Development and Board Member of Apertura. We aim to maximize our impact through dedicated internal programs and meaningful partnerships with other biopharma companies and academic groups. Committed to advancing the field of gene therapy, Apertura seeks to collaborate broadly to accelerate impact to patients.

About Apertura Gene TherapyApertura is a biotechnology company opening opportunities for treating currently intractable diseases. We are uniquely positioned to develop genetic medicines by simultaneously engineering AAV capsids, genetic regulatory elements, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity, and manufacturability. Apertura is committed to growing the field of gene therapy and believes that together we maximize our impact by working with corporate and academic partners, patients, and foundations. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, the company is based at the Cure, Deerfields innovation campus in New York City. For more information, please visit our website at http://www.aperturagtx.com and follow us on LinkedIn and Twitter.

About Deerfield ManagementDeerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, please visit https://deerfield.com/.

About Harvard Universitys Office of Technology DevelopmentHarvards Office of Technology Development (OTD) promotes the public good by fostering innovation and translating new inventions made at Harvard University into useful products that are available and beneficial to society. Our integrated approach to technology development comprises sponsored research and corporate alliances, intellectual property management, and technology commercialization through venture creation and licensing. More than 90 startups have launched to commercialize Harvard technologies in the past 5 years, collectively raising more than $4.5 billion in financing. To further bridge the academic-industry development gap, Harvard OTD manages the Blavatnik Biomedical Accelerator and the Physical Sciences & Engineering Accelerator. For more information, please visit https://otd.harvard.edu.

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Apertura Gene Therapy Launches with $67M Series A Financing from Deerfield and an Innovative Technology Platform to Develop Genetic Medicines -...

Travis Hughes had been assured by both mentors and peers that he was a front-runner for a super-competitive dermatology residency: He had a double degree from Harvard University, experience as a clinical scientist at a health tech startup, and a CV with nearly 40 publications and four patents. Still, during Match Week in March, he failed to land a residency slot, along with 7.1% of fourth-year medical students in the U.S.

While many in his position were upset, Hughes said he was relieved. His part-time job at a startup had nudged him toward industry. The push from the Match was all he needed to set out on this new path. Now, he could forgo the long hours and low pay of residency for a comfortable lifestyle in industry.

From the start of medical school, Hughes had dreamed of eventually founding a biotech company that could sell low-cost drugs, devices, and diagnostics, while he practiced medicine to inform his industry work. His vision to transition from academic medicine to industry put him in good company. Of 2,969 new recipients of a Ph.D. in the life sciences surveyed in 2020, 43% reported that they had committed to a job in industry or business a 10-point jump from 2015, according to the Survey of Earned Doctorates, administered by the National Center for Science and Engineering Statistics. Only 37% reported they would continue down the academic path a 9-point decrease from 2015.

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With both an M.D. and a Ph.D., Hughes is one of those graduates set on a departure from academia. His story provides a window into why so many others have made the same choice.

After dinner one evening in 2020, Hughes wife, Melany Park, newly in her third trimester of pregnancy, said she had an upset stomach. When she went to the bathroom, she realized it was more serious: She was losing blood fast, after her placenta, which had nourished her fetus, had broken off from the inside of her uterus. Park was rushed to the hospital for an emergency C-section, and at 29 weeks, their daughter Isla was born prematurely. She spent the next two months in the intensive care unit.

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Park and their daughter have recovered, but the medical crisis changed how Hughes viewed residency, and how to balance his career ambitions with his family. Isla needs a roster of specialists, including a neonatologist and an occupational therapist, who help ensure that she meets her childhood developmental milestones. So Hughes narrowed his selection for residency to five programs near their home in Massachusetts and in other cities with appropriate early childhood services and where his wife could find work as an architect.

He knew he was taking a risk, especially as a candidate for dermatology, a specialty that nearly 20% of U.S. applicants fail to match into. But now in his mid-30s, with a wife and child to support at home, if he couldnt do residency on his own terms, Hughes was fine with not going at all. The Match system forces desperation in people, he said. I didnt want that lack of control.

Hughes applied to medical school because he wanted to make a direct positive impact on a persons life. As a boy, he watched his father care for his grandmother, who struggled to breathe as emphysema destroyed the airways in her lungs. After he earned his masters degree, in need of a job and a place to live while he applied to medical school, Hughes became an in-home caregiver himself after he responded to an advertisement on Craigslist, posted by a man with multiple sclerosis. In the second year of his doctorate program, Hughes reprised that role, this time with Park, his then-fiance: At Harvard, they lived with and cared for Jack L. Strominger, a renowned immunologist.

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That same desire to help others was what drove him to embark on the long and grueling road of medical training. During his first year at Harvard, he worked in a lab that developed Seq-Well, a cost-effective, portable way to analyze gene expression in cells. With all the tools he needed in a black Pelican case, he traveled to South Africa, the first of many countries on six continents that he would visit to share the technique, which helped galvanize scientific research, especially in places with limited resources.

Inspired by that work, he decided to pursue a doctorate degree in immunology on top of a medical degree. Hughes was encouraged by Strominger to pursue big science questions that fascinated him like how the immune system fights tuberculosis, or how cells mutate in metastatic cancer.

Hughes said that while he enjoyed his time in the lab, fatigue set in: He spent long hours refining lab techniques, writing papers, and traveling to share research globally. As he wearied in the latter phases of his Ph.D., he heard of a company called nference, a local health tech startup. He applied and got a job as a clinical scientist there to help support his family during medical training. It was a breath of fresh air, he said.

He found he was drawn to industry, more so than to working in academic medicine. He felt a weight off his shoulders when he thought of a life free of hustling to publish research, win grants, and earn academic promotions. In exchange for 80-hour work weeks as a resident physician, he envisioned being home to see his toddler grow up. He considered, as well, that an industry salary would provide a far more livable wage: For an entry-level position, he was offered more than four times the median salary of a resident.

Money feels more straightforward, compared to the currencies of authorship, attribution, and credit in academia, said Hughes. In addition, the startup felt team-oriented in a refreshing way. He was especially pleased that the mission of his work, which uses AI to analyze health records, aligned with what motivated him to apply to medical school in the first place: to improve care for patients.

When the news came on the third Monday of March that Hughes had not matched into residency, he felt no pressure to scramble for leftover spots. He was offered an empty position for a clinical scientist at a dermatology residency on the West Coast, but he turned it down. He said he would have felt selfish if he had picked up his family and moved them to the opposite side of the country. He thought of the two paths before him. In industry, he felt he still could make a positive impact that would aid the work of frontline health care workers, and he still could pursue scientific questions that were challenging and exciting. So he chose to transition to a full-time role as a clinical scientist at nference.

For Hughes, no door remains closed, but he says he would be hard-pressed to apply to residency again. He recently caught up with Strominger, both a mentor and the recipient of his care. Uncertain of how Strominger would react, he told him that he planned to pivot to industry. Now retired from working in the lab, Strominger gave his blessing to Hughes for the next step in his journey.

Even in his 90s, you could still find Strominger in the lab, asking new research questions, looking at slides under a microscope, and having his eyes light up when he learned something new. He had a storied career: He won the Lasker Award in 1995 and trained directly or by lineage most of the immunologists at Harvard.

But for Hughes, that sort of career just never felt like exactly what I wanted, he said. Now, I have an opportunity to rethink my life and help others re-envision their path as well.

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He had an M.D. and a Ph.D. but didn't match into a residency. It was the push he needed to jump into health tech - STAT

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Autolus Therapeutics plc (Nasdaq: AUTL) today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead gene therapy obecabatagene autoleucel (obe-cel), a CD19-directed autologous chimeric antigen receptor (CAR) T therapy that is being investigated in the ongoing FELIX Phase 2 study of adult relapsed / refractory B-Acute Lymphocytic Leukemia (ALL).

The FDA grants RMAT designation to drug candidates in recognition of the therapys potential to address significant unmet medical needs in patients with serious or life-threatening conditions. RMAT designation provides important benefits in the drug development process, designed to facilitate and expedite development and regulatory review.

RMAT designation is an important regulatory milestone for obe-cel and highlights its potential to address the unmet medical need for adult patients with relapsed and refractory B-ALL, said Dr. Christian Itin, Chief Executive Officer of Autolus. RMAT designation from FDA, PRIME designation from EMA and ILAP designation from MHRA facilitate regulatory interactions with key health authorities and supports our drive to bring this innovative therapy to patients as quickly as possible.

obe-cel has previously been granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) and Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom.

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Autolus Therapeutics (AUTL) Granted Regenerative Medicine Advanced Therapy Designation to obe-cel - StreetInsider.com

image:Artificial heart tissue can contract against a resistance and then relax. view more

Credit: Michael Gotthardt, MDC

The contractile and elastic properties of the heart are finely tuned. This is a prerequisite for the cardiac cycle and efficient adaptation. At the MDC, Michael Gotthardt investigates the underlying molecular and biomechanical mechanisms. He is awarded with an ERC Advanced Grant for this work.

MERAS is the acronym on the recently approved project proposal. It stands for: Mechanoregulation of alternative splicing. We would like to understand how the heart responds to environmental factors and adjusts its elastic properties such that it can function at an optimal level, says Michael Gotthardt. He heads the research group Neuromuscular and Cardiovascular Cell Biology at the Max Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC). For his project, he now receives an Advanced Grant of 2.5 million from the European Research Council (ERC).

This ERC Advanced Grant is awarded to scientists with more than ten years of research experience who have already played a prominent role in their field. Out of the 1,735 researchers from across Europe who applied for the grants this year, 253 were successful.

The mechanical work of the heart depends on the sarcomere, the smallest contractile unit. Here, actin and myosin filaments facilitate contraction, while the giant protein titin determines the elastic properties of heart muscle cells. Different titin variants (isoforms) are expressed perfectly adapted to the mechanical needs. The researchers wish to investigate the underlying process alternative splicing in detail.

Refined regulatory feedback

Our recent analysis of sarcomeric protein composition not only identified the expected structural proteins, but also proteins that link to cell signalling, metabolism, the regulation of gene expression and alternative splicing. These are proteins you would normally expect in the nucleus but not in the sarcomere, emphasizes Michael Gotthardt. It appears that the sarcomere directly communicates with the nucleus on necessary adaptations. The unexpected feedback from sarcomere to spliceosome could explain how sarcomeres adjust to mechanical stress. This is a new hypothesis that the researchers will explore in depth.

A detailed understanding of the regulatory process could also have therapeutic relevance e.g. for people with heart failure. For most of these patients, the ventricular walls have become so rigid that the chambers are no longer able to fill sufficiently. A drug that interferes with the communication from sarcomere to spliceosome could make a stiff cardiac ventricle more compliant, resulting in more efficient filling.

A second ERC grant

This year, the ERC selection process was not solely based on the submitted proposals, but for the first time included short presentations online of course due to Covid 19. Four slides in eight minutes for a 2.5 million euro project, recounts Michael Gotthardt, who is also Professor of Experimental and translational cardiology at Charit Universittsmedizin Berlin. For the scientist, this is already the second substantial contribution from the EU following his ERC Starting Grant in 2011. It extends over a period of five years. This enables us to build up lasting collaborations and projects with extended time lines. And deep sequencing as a prerequisite to evaluate alternative splicing at scale would otherwise be prohibitively expensive.

Gotthardts team works with genetic mouse models, synthetic heart tissue derived from patient cells and isolated heart muscle cells (cardiomyocytes). Single cell mechanics is precision work. For this, the cardiomyocytes first need to be isolated, secured under a special microscope and electrically stimulated. Then you can derive the active and passive forces, says Michael Gotthardt. This would provide the properties of just one single cell. However, for a convincing study, a large number of these experiments need to be conducted.

The goal: new technologies for single-cell mechanics and multi-omics

Extensive manual work is also required to understand which titin isoforms are expressed in response to stress or disease. Compared to a large piece of tissue, a single cell contains relatively few RNA molecules which means that analysis of gene expression frequently reaches the detection limit.The analysis of alternative splicing is even more difficult for giant titin isoforms with up to 100,000 bases. Here, available short reads need to be assembled like a jigsaw puzzle that misses important pieces, says Michael Gotthardt. With the ERC funding, among other things, he plans to develop technologies for single cell mechanics, -transcriptomics, -proteomics that will facilitate multi-omics approaches and enable higher rates of throughput.

The Max Delbrck Center for Molecular Medicine (MDC)

The Max Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC) is one of the worlds leading biomedical research institutions. Max Delbrck, a Berlin native, was a Nobel laureate and one of the founders of molecular biology. At the MDCs locations in Berlin-Buch and Mitte, researchers from some 60 countries analyze the human system investigating the biological foundations of life from its most elementary building blocks to systems-wide mechanisms. By understanding what regulates or disrupts the dynamic equilibrium in a cell, an organ, or the entire body, we can prevent diseases, diagnose them earlier, and stop their progression with tailored therapies. Patients should benefit as soon as possible from basic research discoveries. The MDC therefore supports spin-off creation and participates in collaborative networks. It works in close partnership with Charit Universittsmedizin Berlin in the jointly run Experimental and Clinical Research Center (ECRC ), the Berlin Institute of Health (BIH) at Charit, and the German Center for Cardiovascular Research (DZHK). Founded in 1992, the MDC today employs 1,600 people and is funded 90 percent by the German federal government and 10 percent by the State of Berlin.www.mdc-berlin.de

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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ERC Advanced Grant for cardiac research at the MDC - EurekAlert

A new version of the Omicron variant of SARS-CoV-2, known as BA.2, has emerged. Although experts are unsure about its effects, they know that it is spreading quickly and has 20 mutations in the area that most COVID-19 vaccines target.

Scientists first identified the BA.2 subvariant of Omicron in India and South Africa in late December 2021. Since then, it has spread to several countries, including the United States, the United Kingdom, and Israel.

The subvariant virus has also spread rapidly in Denmark, increasing from 20% of all COVID-19 cases in the country in week 52 of 2021 to 45% in the second week of 2022.

Despite its rapid spread in the country, initial analyses show no difference in hospitalizations between the BA.2 subvariant and the original form of Omicron, also known as BA.1.

Studies, however, are still ongoing to understand the infectiousness of BA.2, alongside how effective vaccines are against it.

While BA.2 is not currently a variant of concern, public health officials in the U.K. have taken enough interest in its spread to designate it as a variant under investigation.

To understand more about the emerging subvariant, Medical News Today spoke with six experts in public health, immunology, and infectious diseases.

Omicron has three main [subvariants] BA.1, BA.2, and BA.3 according to the World Health Organization (WHO), Dr. Donald C. Vinh, associate professor in the Department of Medicine at McGill University, Canada, told MNT.

Up until now, the overwhelmingly large majority of all Omicron cases has been BA.1. However, in some places, the BA.2 has emerged and has spread faster than BA.1, he went on to note.

This sister variant, which is still Omicron, is interesting because it seems to be displacing Omicron in certain parts of the world, Dr. Amesh A. Adalja, senior scholar at Johns Hopkins Center for Health Security, told MNT. There is speculation that it may be more transmissible than its sibling.

How the fast spread of the BA.2 subvariant may affect public health is still under investigation.

The Omicron SARS-CoV-2 variant has been interesting to scientists because of its comparatively (i) higher number of mutations, which [] allow it to partially evade peoples immune response; (ii) higher transmissibility and pathogenicity, i.e., its greater ability to infect and cause disease; and (iii) lower virulence, i.e., its lower ability to cause severe disease, Dr. Richard Reithinger, Ph.D., vice president of global health at RTI International, explained.

The big question for newly identified variants and subvariants such as omicron BA.2 is how these above three characteristics vary from the original SARS-CoV-2 virus [variant] or the Omicron variant and why.

Dr. Richard Reithinger, Ph.D.

If some countries are now reporting a surge in the proportion of BA.2 subvariant infections, is it because the additional mutations make it more transmissible or allow it to evade the immune response more easily than the other Omicron subvariants? Will BA.2 result in the same clinical pathology as the Omicron parent variant (B.1.1.529) and subvariants (BA.1 and BA.3)? Also, how do current therapeutic options and vaccines fare against BA.2?

According to Dr. Reithinger, these are some of the questions that public health experts must take into consideration while keeping this subvariant under observation.

While researchers are still gathering data on how BA.2 may affect the population at large, laboratory studies have already verified many of its molecular properties.

BA.2 is missing the spike 69-70 mutations, so it does not cause S gene target failure, making it harder to identify on PCR tests, Dr. Anna Ssentongo, assistant professor of public health at the Penn State College of Medicine, told MNT.

Because of this, BA.2 was nicknamed the stealth variant, she explained.

Dr. Ssentongo added that BA.2 has more than 20 mutations in its spike protein, which is a target of many COVID-19 vaccines because the virus uses it to enter healthy cells. Although this difference may make BA.2 more resistant to vaccines, further research is necessary to confirm any effects.

Similar to its parental lineage (Omicron), it is expected to be highly transmissible and result in less severe disease than the Delta or Beta variants, especially if one is fully vaccinated and even better boosted, added Dr. Reithinger. However, ultimately, this would have to be confirmed by ongoing laboratory and clinical studies, which are expected to provide results in the next couple of weeks.

Dr. Vinh agreed that before drawing any conclusions on how the subvariant may affect public health, further research is necessary:

It is important to mention that there are very limited data on clinical differences between BA.2 and BA.1. Specifically, we have no firm data to know if BA.2 is more contagious, results in more severe disease, or can evade immunity better than BA.1. Nonetheless, early data from Denmark and the U.K. suggest that BA.2 may be more contagious than BA.1.

When MNT asked whether we should be concerned about BA.2, Dr. Pavitra Roychoudhury, research associate at the Vaccine and Infectious Disease Division at the University of Washington, explained: It remains to be seen how BA.2 will compete against currently circulating viruses [], and also whether it causes more severe disease. [] We will continue to monitor frequencies of BA.2 through the use of genomic surveillance.

Dr. Adalja agreed that much remains to be seen: We know that this has been a variant that has been present since the early days of Omicron and that it has some similar and some distinct mutations. It is unclear, as of now, whether it is more transmissible. [It] likely has the same characteristics when it comes to vaccine efficacy and severity.

It is too early to know what role [BA.2] may play. Its likely that it will just be part of the Omicron wave and they extend it over time and eventually become the dominant version of Omicron. More study is needed.

Dr. Amesh Adalja

However, Dr. Barton F. Haynes, director of the human vaccine institute in the Department of Medicine at the Duke University School of Medicine, said that due to its molecular changes, there might be cause for concern regarding BA.2.

We are worried that because it is so different than Omicron BA.1, it may escape current vaccines and Omicron BA.1-neutralizing antibodies, he noted. To this point, we are working to study the Omicron BA-2 virus to see whether current vaccine-induced neutralizing antibodies neutralize it.

Whether BA.2, or any other Omicron subvariant or other SARS-CoV-2 variant, there is irrefutable evidence that existing vaccines are quite effective in protecting people against infection and highly effective in protecting people from developing severe disease or worst case scenario dying upon SARS-CoV-2 infection, explained Dr. Reithinger.

Additionally, even if vaccinated, people should consider adhering to nonpharmaceutical interventions, such as face masks, physical distancing, and handwashing, particularly when in crowded and/or high transmission environments, he advised.

For live updates on the latest developments regarding COVID-19, click here.

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Omicron BA.2: Should we be worried? - Medical News Today

News Release

Monday, January 24, 2022

NIH-funded preclinical study identifies potential therapeutic approach to treat ALS.

Using an experimental drug, researchers were able to suppress a mutated amyotrophic lateral sclerosis (ALS) gene. Studies in mice demonstrate that the therapy could show promise in treating rare, aggressive forms of ALS caused by mutations in the fused in sarcoma (FUS) gene. The study, published in Nature Medicine, was funded in part by the National Institute for Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health.

The study models how promising gene-targeting therapies can move expeditiously from pre-clinical development to clinical testing, said Amelie Gubitz, Ph.D., program director at NINDS. There is a desperate need for innovative approaches to treating ALS.

ALS, also known as Lou Gehrigs disease, is a fatal neurological disorder that causes the degeneration of motor neurons in the brain and spinal cord. People with ALS rapidly lose muscle strength and eventually their ability to move, swallow, and breathe. Most cases of ALS are sporadic, but at least 10% of cases are familial, or due to mutations in various genes. Mutations in the gene FUS cause severe forms of ALS, referred to as FUS-ALS, including a rare type that begins in adolescence or young adulthood.

In the study, Neil Shneider, M.D., Ph.D., the Claire Tow Associate Professor of Motor Neuron Disorders and Director of the Eleanor and Lou Gehrig ALS Center at Columbia University, New York City, and his team delayed the onset of motor neuron degeneration in mice by using an antisense oligonucleotide drug designed to silence FUS by blocking cells from making specific proteins. Following encouraging results, they administered the drug to a patient with ALS.

Compared to normal mice, mice with a mutated FUS gene had higher levels of insoluble FUS and other ALS-related proteins in their brains and spinal cords. Mice with higher doses of mutant FUS in motor neurons experienced rapid neurodegeneration that began early in life, much like FUS-ALS patients.

The study establishes a mouse model that is highly disease-relevant, said Dr. Shneider. In mice, we found that FUS toxicity was due to a gain of function and was dose-dependent, suggesting that we could treat FUS-ALS by silencing the FUS gene.

In 2019 Dr. Shneider met an individual with ALS in search of therapies that may help her disease. Inspired by her story, Dr. Shneider teamed up with a pharmaceutical company to develop a personalized therapy designed to target the FUS mutation.

In mice, injecting a single dose of the drug into the ventricles, fluid-filled spaces surrounding the brain, delayed the onset of inflammation and motor neuron degeneration by six months. The drug also knocked down levels of FUS by 50% to 80% in the brain and spinal cord. Following drug administration, insoluble forms of other ALS-associated proteins were also cleared.

Under a compassionate use protocol reviewed by the U.S. Food and Drug Administration, Dr. Shneider administered the drug to the patient it had been designed for. The patient received repeated infusions of the drug into her spinal canal for 10 months. During the treatment, the patients rate of motor function deterioration slowed. The patient tolerated the treatment well and there were no medically adverse effects.

The study is an example of a precision medicine, bench-to-bedside effort, said Dr. Shneider. We began with the mouse model to establish a rationale for the drug, conducted efficacy studies in the mouse, moved the drug into a human, and collected valuable data that was ultimately used to support a larger Phase 3 clinical trial.

Treatment began more than six months after clinical onset, by which time the disease had already significantly advanced. As is typical with juvenile-onset FUS-ALS, the disease progressed rapidly, and the patient died from complications of the disease.

By studying the patients brain and spinal cord tissue, researchers found that the drug silenced FUS throughout the nervous system and reversed the toxic nature of FUS and other disease-related proteins. Compared to tissue from untreated FUS-ALS patients and healthy controls, FUS protein aggregatesa pathological hallmark of this form of ALSwere sparse, suggesting that they may have been cleared by the drug. Tissue samples were provided by the New York Brain Bank of Columbia University.

The protein made from the FUS gene has been shown to be important for various cellular processes. Prior studies in mice suggest that FUS mutations result in the production of an abnormal protein that forms clumps, or aggregates, leading to motor neuron damage. By targeting the faulty gene in a way that suppresses toxic FUS activity, gene silencing products like the antisense oligonucleotide drug could potentially reduce or prevent disease progression.

The results were used to support a clinical trial testing the drug in patients with FUS-ALS (NCT04768972).

This study was supported by grants from the NIH (NS106236), Nancy Perlman, Tom Klingenstein, and the Judith and Jean Pape Adams Charitable Foundation.

NINDSis the nations leading funder of research on the brain and nervous system.The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Korobeynikov, V.A., et al. Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nature Medicine, January 24, 2022. DOI: 10.1038/s41591-021-01615-z

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Silencing a faulty gene may uncover clues to rare forms of ALS - National Institutes of Health

StemExpress to use utilize the Thermo Fisher Accula rapid PCR testing system to provide event attendees with accurate results in 30 minutes.

Published: Oct. 5, 2021 at 3:33 PM EDT|Updated: 2 hours ago

SACRAMENTO, Calif., Oct. 5, 2021 /PRNewswire/ --StemExpress is proud to announce that they will be the official COVID-19 testing provider for 2021's Meeting on the Mesa, a hybrid event bringing together great minds in the cell and gene biotech sphere. It has partnered with Alliance for Regenerative Medicine to comply with the newly implemented California state COVID-19 vaccination and testing policy regarding gatherings with 1,000 or more attendees. This partnership will allow the vital in-person networking aspect of the event to commence while protecting the health and safety of participants and attendees.

In-person networking commences at the 2021 Cell and Gene Meeting on the Mesa with COVID-19 testing options provided by StemExpress.

As a leading global provider of human biospecimen products, StemExpress understands the incredible impact that Meeting on the Mesa has on the industry and has been a proud participant for many years. For over a decade, StemExpress has provided the cell and gene industry with vital research products and holds valued partnerships with many of this year's participants. As such, it understands the immense value that in-person networking provides and is excited to help bring this element back to the meeting safely and responsibly.

StemExpress has been a trusted provider of widescale COVID-19 testing solutions since early 2020 - providing testing for government agencies, public health departments, private sector organizations, and the public nationwide. For Meeting on the Mesa, StemExpress is offering convenient testing options for unvaccinated attendees and those traveling from outside of the country. Options will include take-home RT-PCR COVID Self-Testing Kits and on-site, rapid PCR testing for the duration of the event. The self-testing kit option allows attendees to test for COVID in the days leading up to the event for a seamless admission and the days following the event to confirm they haven't been exposed. The on-site rapid testing option utilizes the new Thermo Fisher Accula, offering in-person testing at the event with results in around 30 minutes. StemExpress is excited to bring these state-of-the-art COVID testing solutions to the frontlines of the Cell & Gene industry to allow for safe in-person connections.

The StemExpress partnership with Alliance for Regenerative Medicine seeks to empower the entire cell and gene industry with a long-awaited opportunity to return to traditional networking practices. It is well known that innovation doesn't exist in a vacuum - allowing great minds to come together is a sure way to spur scientific growth and advance cutting-edge research, giving hope for future cures.

Cell and Gene Meeting on the Mesa will take place October 12th, 2021, through October 14th, 2021, at Park Hyatt Aviara,7100 Aviara Resort Drive Carlsbad, CA 92011. To learn more about the event, please visit MeetingOnTheMesa.com.

For more information about COVID testing solutions for businesses and events, visit https://www.stemexpress.com/covid-19-testing/.

About StemExpress:

Founded in 2010 and headquartered in Sacramento, California, StemExpress is a leading global biospecimen provider of human primary cells, stem cells, bone marrow, cord blood, peripheral blood, and disease-state products. Its products are used for research and development, clinical trials, and commercial production of cell and gene therapies by academic, biotech, diagnostic, pharmaceutical, and contract research organizations (CRO's).

StemExpress has over a dozen global distribution partners and seven (7) brick-and-mortar cellular clinics in the United States, outfitted with GMP certified laboratories. StemExpress runs its own non-profit supporting STEM initiatives, college and high school internships, and women-led organizations. It is registered with the U.S. Food and Drug Administration (FDA) and is continuously expanding its network of healthcare partnerships, which currently includes over 50 hospitals in Europe and 3 US healthcare systems - encompassing 31 hospitals, 35 outpatient facilities, and over 200 individual practices and clinics.

StemExpress has been ranked by Inc. 500 as one of the fastest-growing companies in the U.S.

About the Alliance for Regenerative Medicine:

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 400+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Media Contact: Anthony Tucker, atucker@stemexpress.com

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - WITN

UMass Amherst pre-veterinary sciences major uses gene sequencing to advance immunology research

As a young girl dreaming of becoming a veterinarian, Kate Loonie may have expected some of the hands-on experiences she had at UMass Amherst. She vaccinated and ear-tagged Belted Galloway cattle, medicated injured horses, helped deliver lambs, and even cared for turtles as a summer intern at the New England Aquarium. But she didnt expect that some of her most fulfilling work would be performing gene sequencing on a computer.

I never saw myself doing research, especially in my undergraduate years, Kate says. I didnt think I would enjoy lab work. But it pushed me to places I didnt think Id be able to go.

Working in the lab of Professor of Veterinary and Animal Sciences Cynthia Baldwin beginning in her freshman year, Kate has been involved in three cellular immunology research projects related to tracking down a gene family, known as WC1, in sheep, cattle, and goats. She graduated from UMass with her name on three published research papers.

Although genetic sequences at first looked like alphabet soup to her, Kate learned the Baldwin labs bioinformatics technology quickly. I practiced and practiced that tedious software every moment I could, she recalls. Baldwin noted that Kate is a good collaborator who is proactive in the lab. She is really passionate about learning new techniques and is a very mature, independent, and responsible individual, she said.

Having learned the software, Kate was given an independent projectlooking at the gene sequences of the auroch, the ancient ancestors of our modern-day cattle. The auroch, immortalized in cave paintings, were massive super cattle that lived in Asia, Europe, and North Africa before their extinction in 1627. Kate sequenced the DNA from a 6,000 year-old auroch humerus bone and gathered evidence of the presence of WC1. I concluded that some bovine WC1 molecules were preserved from the ancient auroch, while some molecules seem to have been lost or created from evolution. This shows its possible the aurochs were seeing the same pathogens as were seeing today, she says. This research has important implications for improving the health of ruminants.

For her second published project, Kate amalgamated data from numerous years of study and used gene sequencing technology to investigate WC1 in sheep. Her third published paper is titled, Special features of T cells in ruminants. Kates thesis, Investigating gene expression in T cells, contributes toward understanding cells that could point the way toward more effective vaccines.

For Kate, lab work revealed the true potential of the impact a veterinarian can have in research. Research made me stop and think, she says. I started to think about situations differently and more analytically both in the lab and out of the lab, in classes, at the cattle barn, and everywhere else. I caught a glimpse of the potential I will have with more education, and how much I can contribute to developing science and knowledge in my future. With that, I relit my passion for veterinary science.

Kate will begin studying for her doctor in veterinary medicine degree at Cornell University this summer. After I have established my veterinary career, she says, I can see myself pursuing a masters or PhD in the research realm. I hope to teach the young undergraduate generation of pre-veterinary students.

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Kathleen Loonie '21: Rising Researcher - UMass News and Media Relations

This article was originally published here

Sci Rep. 2022 Jan 27;12(1):1442. doi: 10.1038/s41598-021-03324-6.

ABSTRACT

Perforin secreted from cytotoxic lymphocytes plays a critical role in cancer immunosurveillance. The aim of this study was to investigate the therapeutic potential of liposomes containing perforin expression vector driven by the promotor of prostate-specific antigen (PSA). The anti-tumor effect of perforin was analyzed using prostate cancer (PC) PC-3 cells in which perforin expression was controlled by Tet-on system (PC-3PRF cells). Liposomes encapsulating PSA promoter-driven perforin expression vector (pLipo) were constructed for its specific expression in PC. The anti-tumor effect of pLipo was evaluated in vitro using docetaxel-resistant PC 22Rv1 PC cell line, 22Rv1DR, and PC-3 cells in the presence of human peripheral blood mono nuclear cells (PBMCs) and also in vivo using male nude mice bearing 22Rv1DR cell-derived tumor xenograft. Induction of perforin significantly inhibited growth of PC-3PRF cells. Treatment with pLipo induced perforin expression in 22Rv1DR cells expressing PSA but not in PC-3 cells lacking it. Treatment with pLipo at a low concentration was prone to inhibit growth of both cell lines and significantly inhibited growth of 22Rv1DR cells when co-incubated with PBMCs. The combined use of pLipo at a high concentration with PBMCs showed nearly complete inhibition of 22Rv1DR cell growth. Intravenous administration of pLipo via tail vein increased the level of perforin in tumor and serum and significantly decreased the tumor volume. Our results suggest that liposome-mediated PC-specific expression of perforin could be a novel therapy for advanced PC.

PMID:35087064 | DOI:10.1038/s41598-021-03324-6

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Gene therapy of prostate cancer using liposomes containing perforin expression vector driven by the promoter of prostate-specific antigen gene -...

How often a person takes daytime naps, if at all, is partly regulated by their genes, according to new research led by investigators at Harvard-affiliated Massachusetts General Hospital (MGH) and published inNature Communications.

In this study, the largest of its kind ever conducted, the MGH team collaborated with colleagues at the University of Murcia in Spain and several other institutions to identify dozens of gene regions that govern the tendency to take naps during the day. They also uncovered preliminary evidence linking napping habits to cardiometabolic health.

Napping is somewhat controversial, says Hassan Saeed Dashti of the MGH Center for Genomic Medicine, co-lead author of the report with Iyas Daghlas, a medical student at Harvard Medical School (HMS). Dashti notes that some countries where daytime naps have long been part of the culture (such as Spain) now discourage the habit. Meanwhile, some companies in the United States now promote napping as a way to boost productivity. It was important to try to disentangle the biological pathways that contribute to why we nap, says Dashti.

Previously, co-senior author Richa Saxena, principal investigator at the Saxena Lab at MGH, and her colleagues used massive databases of genetic and lifestyle information to study other aspects of sleep. Notably, the team has identified genes associated with sleep duration, insomnia, and the tendency to be an early riser or night owl. To gain a better understanding of the genetics of napping, Saxenas team and co-senior author Marta Garaulet of the department of physiology at the University of Murcia, performed a genome-wide association study (GWAS), which involves rapid scanning of complete sets of DNA, or genomes, of a large number of people. The goal of a GWAS is to identify genetic variations that are associated with a specific disease or, in this case, habit.

For this study, the MGH researchers and their colleagues used data from the UK Biobank, which includes genetic information from 452,633 people. All participants were asked whether they nap during the day never/rarely, sometimes or usually. The GWAS identified 123 regions in the human genome that are associated with daytime napping. A subset of participants wore activity monitors called accelerometers, which provide data about daytime sedentary behavior, which can be an indicator of napping. This objective data indicated that the self-reports about napping were accurate. That gave an extra layer of confidence that what we found is real and not an artifact, says Dashti.

Several other features of the study bolster its results. For example, the researchers independently replicated their findings in an analysis of the genomes of 541,333 people collected by 23andMe, the consumer genetic-testing company. Also, a significant number of the genes near or at regions identified by the GWAS are already known to play a role in sleep. One example isKSR2, a gene that the MGH team and collaborators had previously found plays a role in sleep regulation.

Digging deeper into the data, the team identified at least three potential mechanisms that promote napping:

This tells us that daytime napping is biologically driven and not just an environmental or behavioral choice, says Dashti.

Some of these subtypes were linked to cardiometabolic health concerns, such as large waist circumference and elevated blood pressure, though more research on those associations is needed.

Future work may help to develop personalized recommendations for siesta, says Garaulet.

Furthermore, several gene variants linked to napping were already associated with signaling by a neuropeptide called orexin, which plays a role in wakefulness. This pathway is known to be involved in rare sleep disorders like narcolepsy, but our findings show that smaller perturbations in the pathway can explain why some people nap more than others, says Daghlas.

Saxena is the Phyllis and Jerome Lyle Rappaport MGH Research Scholar at the Center for Genomic Medicine and an associate professor of anesthesia at HMS.

The work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Heart, Lung, and Blood Institute, MGH Research Scholar Fund, Spanish Government of Investigation, Development and Innovation, the Autonomous Community of the Region of Murcia through the Seneca Foundation, Academy of Finland, Instrumentarium Science Foundation, Yrj Jahnsson Foundation, and Medical Research Council.

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The science behind those afternoon naps Harvard Gazette - Harvard Gazette

HILDEN, Germany & GERMANTOWN, Md.--(BUSINESS WIRE)--QIAGEN N.V. (NYSE:QGEN; Frankfurt Prime Standard:QIA) today announced a global collaboration with Mirati Therapeutics Inc. (NASDAQ:MRTX) to continue developing a tissue-based KRAS companion diagnostic to identify patients with cancers that have a KRASG12C mutation who may benefit from treatment with adagrasib, Miratis investigational, highly selective and potent oral small molecule inhibitor of KRASG12C.

The agreement initially focuses on a companion diagnostic test for non-small cell lung cancer (NSCLC), and allows for further development of tests for other Mirati oncology programs.

The planned companion diagnostic would expand upon QIAGENs therascreen KRAS testing portfolio based on real-time qualitative PCR for the QIAGEN Rotor-Gene Q MDx instrument, a member of the modular QIAsymphony family of automation solutions, and builds upon the Companys nearly decade of experience in KRAS companion diagnostic test development and commercialization. QIAGEN and Mirati have previously partnered for the development of a companion diagnostic.

We are pleased Mirati recognizes the success of QIAGENs therascreen platform and continues to partner with us to develop a tissue-based companion diagnostic to identify patients who may benefit from adagrasib. QIAGENs experience and expertise in developing diagnostic solutions for Precision Medicine are well-suited to evaluate patients with non-small cell lung cancer, said Jean-Pascal Viola, Senior Vice President and Head of QIAGENs Molecular Diagnostics Business Area. Our collaboration with Mirati is a demonstration of QIAGENs capabilities as a preferred partner of pharmaceutical and biotech companies for the creation of companion diagnostics.

The therascreen-based companion diagnostic detects KRASG12C, a genetic mutation that is one of the most common KRAS alterations linked to cancer. The RAS gene family is the most frequently mutated oncogene in human cancer, with KRAS being the most prevalent driver mutation in NSCLC.

QIAGEN is a pioneer in Precision Medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from next-generation sequencing (NGS) to polymerase chain reaction (PCR) for companion diagnostic development. QIAGEN has nine PCR based companion diagnostics that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer.

Currently, QIAGEN is working under master collaboration agreements with more than 25 companies to develop and commercialize companion diagnostic tests for their drug candidates a deep pipeline of potential future products to advance Precision Medicine for the benefit of patients. QIAGEN is partnering with Illumina to broaden the availability and use of NGS-based in-vitro diagnostic (IVD) kits, including companion diagnostics, for patient management.

About QIAGEN

QIAGEN N.V., a Netherlands-based holding company, is the leading global provider of Sample to Insight solutions that enable customers to gain valuable molecular insights from samples containing the building blocks of life. Our sample technologies isolate and process DNA, RNA and proteins from blood, tissue and other materials. Assay technologies make these biomolecules visible and ready for analysis. Bioinformatics software and knowledge bases interpret data to report relevant, actionable insights. Automation solutions tie these together in seamless and cost-effective workflows. QIAGEN provides solutions to more than 500,000 customers around the world in Molecular Diagnostics (human healthcare), Applied Testing (primarily forensics), Pharma (pharma and biotech companies) and Academia (life sciences research). As of March 31, 2020, QIAGEN employed approximately 5,700 people in over 35 locations worldwide. Further information can be found at http://www.qiagen.com.

Forward-Looking Statement

Certain statements contained in this press release may be considered forward-looking statements within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended. To the extent that any of the statements contained herein relating to QIAGEN's products, collaborations markets, strategy or operating results, including without limitation its expected adjusted net sales and adjusted diluted earnings results, are forward-looking, such statements are based on current expectations and assumptions that involve a number of uncertainties and risks. Such uncertainties and risks include, but are not limited to, risks associated with management of growth and international operations (including the effects of currency fluctuations, regulatory processes and dependence on logistics), variability of operating results and allocations between customer classes, the commercial development of markets for our products to customers in academia, pharma, applied testing and molecular diagnostics; changing relationships with customers, suppliers and strategic partners; competition; rapid or unexpected changes in technologies; fluctuations in demand for QIAGEN's products (including fluctuations due to general economic conditions, the level and timing of customers' funding, budgets and other factors); our ability to obtain regulatory approval of our products; difficulties in successfully adapting QIAGEN's products to integrated solutions and producing such products; the ability of QIAGEN to identify and develop new products and to differentiate and protect our products from competitors' products; market acceptance of QIAGEN's new products and the integration of acquired technologies and businesses. For further information, please refer to the discussions in reports that QIAGEN has filed with, or furnished to, the U.S. Securities and Exchange Commission (SEC).

Category: Corporate

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QIAGEN Partners With Mirati Therapeutics Inc. to Develop KRASG12C Companion Diagnostic for Non-Small Cell Lung Cancer (NSCLC) - Business Wire

ALISO VIEJO, Calif., Feb. 10, 2021 /PRNewswire-PRWeb/ -- RARE-X today announced three new board members who will help support the nonprofit's work in structured patient data collection, responsible data sharing, and the promise of its Federated Data Sharing Platform for data sharing and analysis. The new board members are Cynthia Grossman, PhD, director at Biogen; Jason Colquitt, CEO of Across Healthcare; and Simon Frost, CEO of Tiber Capital Group.

"The additions of Cynthia Grossman, PhD, Jason Colquitt, and Simon Frost to the board are very strategic. All bring a depth of knowledge in patient advocacy, health tech, scaling-up organizations, and operational excellence," said Nicole Boice, RARE-X Co-Founder/Executive Director. "We are honored to have them join an already extraordinary board and thrilled to channel their expertise, talent, and energy into helping RARE-X build towards the future."

Cynthia Grossman, PhD, is a director at Biogen, leading the MS PATHS program, a collaborative research network aimed at generating evidence to improve outcomes for patients living with Multiple Sclerosis. Prior to joining Biogen, Cynthia was director at FasterCures, a center of the Milken Institute. Before joining FasterCures, she was chief of the HIV Care Engagement and Secondary Prevention Program in the Division of AIDS Research (DAR) at the National Institute of Mental Health (NIMH). Cynthia has spent her career working to improve health by expanding opportunities for patients' perspectives to shape the processes by which new therapies are discovered, developed, and delivered. Cynthia graduated Phi Beta Kappa from Earlham College with a B.A. in psychology and biology and earned her Ph.D. in clinical psychology from the University of Vermont. She has been the recipient of a National Science Foundation Incentives for Excellence Scholarship, an NIH Ruth L. Kirschstein National Research Services Award, and a Postdoctoral Fellowship in Pediatric Psychology at the Warren Alpert Medical School of Brown University.

Jason Colquitt is CEO of Across Healthcare, a company he founded in 2012, leveraging his 20+ years in the healthcare technology field. His work has caused positive disruption within the healthcare industry as he has partnered with many organizations ranging from small start-ups to some of the world's largest health companies including Greenway Health, Walgreens Boots Alliance, Quintiles, IQVIA, Cystic Fibrosis Foundation, Muscular Dystrophy Association, American College of Surgeons, and American Heart Association. Jason has worked directly with patients, caregivers, physicians, regulators, and researchers. Jason was diagnosed with Carnitine Palmitoyltransferase II Deficiency (CPT II), a rare mitochondrial disease. He has used his experiences and technical background to help the rare disease community. Jason holds a Bachelor's degree in Applied Mathematics from Auburn University.

Simon Frost is the CEO of Tiber Capital Group. Before joining Tiber Capital Group, he was the chief investment officer of Greencourt Capital, a public company with approximately $1 billion in real estate assets. Before joining Greencourt Capital, Simon was president and COO of Key Properties. He was also the co-founder of The American Home, one of the largest single-family rental aggregators in the United States. Simon holds Bachelor's and Master's degrees in economics from Cambridge University in England, and a Bachelor's degree in finance from the University of South Africa. Simon serves as director of both Cure AHC and Hope For Annabel, charities dedicated to finding therapies for Alternating Hemiplegia of Childhood.

The current RARE-X Board of Directors includes: Betsy Bogard, head of program and alliance management within the 4:59 Initiative at 5AM Ventures; Nicole Boice, co-founder and executive director of RARE-X; Jason Colquitt, CEO of Across Healthcare; Wendy Erler, vice president of Patient Experience, STAR and Advocacy at Alexion Pharmaceuticals; Simon Frost, CEO of Tiber Capital Group; Peter Goodhand, CEO of Global Alliance for Genomics and Health; Cynthia Grossman, PhD, director at Biogen; Walt Kowtoniuk, PhD, COO of MOMA Therapeutics and venture partner at Third Rock Ventures; Craig Martin, president of Rithm Health and interim CEO at Global Genes; Katherine Maynard, principal at PWR; Angeli Moeller, PhD, head of Pharma Informatics International at Roche; David Pearce, PhD, president of Innovation and Research for Sanford Health; Anthony Philippakis, MD, PhD, chief data officer at Broad Institute; John Reynders, PhD, chief data scientist at Reynders Consulting; Morrie Ruffin, co-founder and board member of ARM Foundation for Cell and Gene Medicine and managing partner, Adjuvant Partners; Alvin Shih, MD, president and CEO at Catamaran Bio.

ABOUT RARE-X

RARE-X is a 501(c)(3) patient advocacy organization focused on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by rare disease. Enabled by best-in-class technology, patients, researchers, and other technology vendors, RARE-X will gather structured, fit-for-purpose data to share broadly, benefitting from 21st-century governance, consent, and federated data sharing technology. RARE-X is building the largest collaborative patient-driven, open-data access project for rare diseases globally. For more information, visit http://www.rare-x.org.

Media Contact:

Tom Hume, Marketing Communications RARE-X

tomh@rare-x.org

Media Contact

Tom Hume, RARE-X, 7602144863, tomh@rare-x.org

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RARE-X Announces the Expansion of its Board of Directors to Support the Organization's Growth and Launch Efforts - WFMZ Allentown

Precision medicine and genetic testing will reign supreme when it comes to the future of Alzheimer disease care and treatment advancement, according to Richard S. Isaacson, MD, director of the Alzheimer's Prevention Clinic and associate professor of Neurology at Weill Cornell Medicine and New York-Presbyterian.

Isaacsons session Treatment Updates in Alzheimer's Disease: Tailoring Management and Care Approaches to Improve Outcomes, was presented atthis year'sNational Association of Managed Care Physicians Spring Managed Care Forum, held in Orlando, Florida, April 21-22.

Kicking off his presentation, Isaacson remarked on the recent FDA approval and CMS coverage determination of Aduhelm (aducanumab), the first new treatment for the disease brought to market in nearly 2 decades. The treatment functions by targeting the buildup of amyloid beta plaques in the brain, which are thought to be a pathophysiological driver of the neurodegenerative disease.

Following its controversial launch, CMS announced it would only cover administration if the drug were delivered in a clinical trial setting.

Despite the attention that development garnered from the press and public alike, Isaacson stressed a lesser-known development in Alzheimer disease detectionthat a blood-based biomarker for amyloid exists, offering an alternative to testing of cerebral spinal fluid.

Most people are unaware of [the blood-based biomarker], he said, equating the advancement to a cholesterol test for the brain.

Diagnostic testing is important because even though individuals may exhibit symptoms of the disease at age 85, the disease first starts in the brain at age 55 or 60. It begins in the brain 20 to 30 years before the first symptom of memory loss begins, providing a wide window of potential intervention time, Isaacson explained.

The disease is also a spectrum, based on the presentation of certain clinical symptoms over decades, he stressed. Individuals can have amyloid present in the spinal fluid for years and not exhibit symptoms, with recent data showing the amyloid levels in the blood actually increase prioir to those in spinal fluid.

Alzheimers disease affects 46 million Americans, Isaacson said. Not all of those people are going to get dementia because they're going to die from something else.

With regard to genetic testing, studies have shown presence of the APOE4 gene may increase individuals risk of dementia. However, if individuals have multiple copies of the APOE variantand 10% of the Alzheimer disease population doesthey are at an increased risk of side effects for aducanumab, underscoring the importance of precision medicine.

Genetic testing is controversial, Isaacson said, but the practice opens up a realm of possibilities for precision medicine for those with the highly heterogenous disease.

Lots of different genes can contribute to development of Alzheimer disease, and based on these, patients can and should receive different treatments, he stressed. In a few years, Isaacson expects computer programs to be able to determine polygenetic risk for the disease, taking these genetic factors into account as opposed to singular variants in isolation. But just because an individual is at risk does not mean they will develop the disease.

Another aspect of developing personalized care for this patient population is tracking of bodily metrics like blood sugar, as research has proved poor blood sugar control is associated with cognitive problems.

Currently, no curative medications for Alzheimer disease exist, only disease modifying therapies which provide modest benefits to patients. For these, Isaacson stressed the notion of start low, go slow, to help mitigate or even avoid any potential adverse effects. However, as more and more therapeutic targets are identified in the research, the potential for new treatment grows.

With regard to preventive measures, Isaacson highlighted the protective effects of the Mediterranean diet, regular exercise and sleep schedules, and low levels of stress and blood pressure. Cognitive engagement activities like listening to and practicing music can also help prevent decline.

Although the metabolic benefits of medications like semaglutide have been proven, and this could in turn help reduce Alzheimer disease risk, Isaacson prefers to recommend his patients follow behavioral changes and use devices like continuous glucose monitors to help assess metabolic metrics.

Overall, precision medicine and vascular risk factor care will kind of turn Alzheimer's disease and cognitive decline on its head. I think were just a couple of years away from that, he said.

Based on modifiable risk factors, 4 out of every 10 cases of Alzheimer disease may be preventable, he concluded, but cautioned that even though someone may do everything right, they can still get the disease.

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The Promise of Precision Medicine and Genetic Testing in Alzheimer Disease - AJMC.com Managed Markets Network

MarketandResearch.biz has brought the addition of a new report examination on Global Gene Therapy Medicine Market affords detailed coverage of the agency and major market trends with ancient and forecast market data. Furthermore, the report gives thorough research into the local improvements of the market, influencing its improvement all through the forecast period from 2022 to 2028.

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At first, the document gives an essential define of the commercial agency that covers definitions and applications. The record profiles the crucial members in the business, along with an itemized analysis in their positions towards the global landscape. The file gives the distinctive study for employer profiling of key market players in the global Gene Therapy Medicine market and comparative analysis based on their product offering, business overviews, geographic presence, commercial enterprise strategies, mergers & acquisitions, SWOT analysis, latest developments, and key financial facts.

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An agreed Paediatric Investigation Plan (PIP) is the regulatory pathway to market authorization for leniolisib as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in children

A Promising Innovative Medicine (PIM) designation is an early indication that leniolisib is a candidate for the MHRA's Early Access to Medicines Scheme

LEIDEN, the Netherlands, April 26, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or the "Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that it has received a positive decision from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) on a Paediatric Investigation Plan (PIP) submission for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3K) inhibitor, for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients from 1 year of age to less than 18 years of age. The Company also announces that the MHRA has granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS.

A PIP is a development plan aimed at ensuring that the necessary data are obtained to support the marketing authorization of a medicine in the pediatric population. All applications for marketing authorization for new medicines in children require the results of studies as described in an agreed PIP, unless the medicine is exempt due to a deferral or waiver. The leniolisib PIP includes two planned global clinical trials in pediatric patients with APDS, the first in children ages 4-11 years and the second in children ages 1-6 years. The Company expects to initiate recruitment for this pediatric program for leniolisib during the second half of 2022.

A PIM designation indicates that a medicinal product is a promising candidate for the MHRA's Early Access to Medicines Scheme (EAMS), which provides pre-market access to products that are intended for the treatment, diagnosis, or prevention of a life-threatening or seriously debilitating condition and have the potential to address an unmet medical need.

Anurag Relan MD, Pharming's Chief Medical Officer, commented:

"We are pleased to have received agreement of this PIP and the PIM designation from the MHRA, these represent important regulatory milestones as we continue to advance leniolisib for the treatment of APDS, a rare, often debilitating, and sometimes fatal condition. The agreed PIP and the PIM designation further support our confidence in the potential of leniolisib to address APDS, as they provide us with a pathway toward marketing authorization in the United Kingdom. Pharming looks forward to continuing to work with regulatory authorities globally to bring leniolisib to patients with this significant unmet medical need as expeditiously as possible."

Leniolisib, which has received Orphan Drug Designation in Europe, received the same designation from the US Food and Drug Administration on January 30, 2018.

As previously announced, Pharming plans to begin submitting global registration filings for leniolisib in the second quarter of 2022 and, subject to approval, launching the treatment in the US in the first quarter of 2023 and starting a series of European launches in the second half of 2023.

About Activated Phosphoinositide 3-Kinase Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately one to two people per million. Also known as PASLI, it is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3K (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3K pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.

About leniolisib

Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3K and PI3K, which are ubiquitously expressed, PI3K and PI3K are expressed primarily in cells of hematopoietic origin. The central role of PI3K in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3K is a valid and potentially effective therapeutic target for several immune diseases.

To date, leniolisib has been well tolerated during both a Phase 1 first-in-human trial in healthy subjects and a Phase II/III registration-enabling study.

About Pharming Group N.V.

Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit http://www.pharming.com.

Forward-looking Statements

This press release contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2021 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2021 filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release.

References:

1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.

2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.

3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.

4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.

5. Maccari ME, et al. Front Immunol. 2018;9:543.

6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.

7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338

For further public information, contact:Pharming Group, Leiden, The NetherlandsSijmen de Vries, CEO: T: +31 71 524 7400 E: [emailprotected]

FTI Consulting, London, UK Victoria Foster Mitchell/Alex ShawT: +44 203 727 1000

FTI Consulting, USA Jim PolsonT: +1 (312) 553-6730

LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon MelensT: +31 6 53 81 64 27E: [emailprotected]

US PR:Emily VanLareE: [emailprotected]T: +1 (203) 985 5596

EU PR:Dan CaleyE: [emailprotected]T: +44 (0) 787 546 8942

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Pharming receives agreement of Paediatric Investigation Plan and Promising Innovative Medicine designation for leniolisib from UK MHRA - PR Newswire

RenaCARE study expected to be fully enrolled by end of Q2 of 2022

AUSTIN, Texas, April 26, 2022 /PRNewswire/ -- Natera, Inc.(NASDAQ: NTRA), a global leader in cell-free DNA testing, today announced the RenaCARE (Renasight Clinical Application, Review and Evaluation) study - a real world, prospective, multi-center clinical study to assess the clinical utility of the Renasight genetic testing panel. The study has already enrolled 1,600 patients across 25 sites, representing leading academic and private nephrology clinics in the U.S., and will enroll up to 2,000 patients. It is expected to complete enrollment in the second quarter of 2022 with a publication expected to be submitted in late 2022.

The study aims to demonstrate how genetic findings impact the management of patient care and examines diagnostic outcomes of patients tested with the Renasight genetic testing panel. In addition, the study will assess patient satisfaction, health knowledge and genetic literacy. This study follows a 2019 publication1 in The New England Journal of Medicine (NEJM) showing that 89% of patients who tested positive with a multi-gene genetic test had actionable clinical implications.

"Chronic kidney disease affects more than 10% of the global population, and our 2019 NEJM study showed roughly a 10% genetic yield among CKD patients," said Ali Gharavi, M.D., chief of the Division of Nephrology at New York-Presbyterian/Columbia University Irving Medical Center, director of the Center for Precision Medicine and Genomics in the Department of Medicine, interim director of the Institute of Genomic Medicine at Columbia University Vagelos College of Physicians and Surgeons, the study's principal investigator and a close collaborator with Kidney Disease: Improving Global Outcomes foundation (KDIGO) and the National Kidney Foundation (NKF). "We're optimistic that this study will show that next-generation sequencing (NGS) multi-gene assays can be used in a real world setting, to inform and guide disease management and help improve patient outcomes."

"We're confident that the RenaCARE study will confirm the high clinical utility shown in prior studies. This will be an important addition to the growing body of evidence showing the value of genetic testing to clarify an undifferentiated diagnosis, identify a genetic subtype within a diagnosis, reclassify a diagnosis, or provide insights for genetic counseling, family planning and clinical trial access," said Hossein Tabriziani, M.D., senior medical director of organ health for Natera.

All patients undergoing testing using the Renasight panel are offered optional pre- and post-test genetic information sessions with a genetic counselor in addition to their test results. Similarly, providers have access to Natera's genetic counselors for questions about the Renasight testing panel and review of test results.

Natera designed and launched the Renasight genetic testing panel with the feedback of general nephrologists, pediatric nephrologists, and transplant nephrologists. Natera has performed over 10,000 Renasight tests to date.

About Renasight

The Renasight test is a germline genetic test that screens for hereditary causes of kidney disease. It is indicated for patients with diagnosed kidney disease and is run from a patient's blood or saliva sample. Providers can use the Renasight test to identify a genetic predisposition, clarify a clinical diagnosis, or identify the etiology of an unknown kidney disease to help inform medical management. Additionally, genetic counseling and familial testing can be offered based on the test result. The test has been developed and its performance characteristics determined by the CLIA-certified laboratory performing the test. The test has not been cleared or approved by the U.S. Food and Drug Administration (FDA). CAP accredited, ISO 13485 certified, and CLIA certified.

About Natera

Natera is a global leader in cell-free DNA testing, dedicated to oncology, women's health, and organ health. Our aim is to make personalized genetic testing and diagnostics part of the standard of care to protect health and enable earlier and more targeted interventions that help lead to longer, healthier lives. Natera's tests are validated by more than 100 peer-reviewed publications that demonstrate high accuracy. Natera operates ISO 13485-certified and CAP-accredited laboratories certified under the Clinical Laboratory Improvement Amendments (CLIA) in Austin, Texas and San Carlos, California. For more information, visit http://www.natera.com.

Forward-Looking Statements

All statements other than statements of historical facts contained in this press release are forward-looking statements and are not a representation that Natera's plans, estimates, or expectations will be achieved. These forward-looking statements represent Natera's expectations as of the date of this press release, and Natera disclaims any obligation to update the forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual results to differ materially, including with respect to whether the results of clinical or other studies will support the use of our product offerings, our expectations of the reliability, accuracy and performance of our screening tests, or of the benefits of our screening tests and product offerings to patients, providers and payers. Additional risks and uncertainties are discussed in greater detail in "Risk Factors" in Natera's recent filings on Forms 10-K and 10-Q and in other filings Natera makes with the SEC from time to time. These documents are available atwww.natera.com/investorsandwww.sec.gov.

ContactsInvestor Relations:Mike Brophy, CFO, Natera, Inc., 510-826-2350Media:Kate Stabrawa, Communications, Natera, Inc., 720-318-4080pr@natera.com

References

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Natera Announces Definitive Study to Evaluate the Clinical Utility of Renasight in the Diagnosis and Management of Chronic Kidney Disease (CKD) -...

After age and genetics, being a woman is the single most important risk factor for developing Alzheimer's disease, experts say.

"Two out of every three brains affected by Alzheimer's disease are women's brains," said Dr. Richard Isaacson, director of the Alzheimer's Prevention Clinic in the Center for Brain Health at Florida Atlantic University's Schmidt College of Medicine.

Now, a new study has good news when it comes to giving women a chance to reduce their increased risk. Personalized lifestyle interventions -- such as diet, exercise, stress reduction and sleep hygiene -- were able to reduce Alzheimer's risk factors in both sexes, but they worked even better in women.

"Our individually tailored interventions led to greater improvements in women compared to men across risk scales for Alzheimer's and cardiovascular disease," said Isaacson, who coauthored the paper.

Women also showed greater improvements than men in biomarkers such as lower blood sugar and lower LDL, or low-density lipoprotein, which is the "bad" cholesterol.

"This study clearly reinforces the need for additional larger studies to be able to better predict the baseline cognitive trajectory in aging females versus males," said Rudy Tanzi, a professor of neurology at Harvard Medical School. He is director of the genetics and aging research unit at Massachusetts General Hospital in Boston.

"As we aim to find ways to nip this disease in the bud stage, we will need to know if prevention and treatment strategies will work equivalently on both men and women. This new study clearly brings us a big step closer to that goal," said Tanzi, who was not involved in the study.

The new study followed a subset of people participating in a 10-year study designed to test the impact of personalized recommendations on cognitive function and risk factors for dementia. The Comparative Effectiveness Dementia and Alzheimer's Registry trial, which began in 2018, is being conducted at NewYork-Presbyterian/Weill Cornell Medical Center in New York City.

After full blood, physical, cognitive and genetic workups, patients were provided with individualized genetic counseling and education. Medications, vitamins and supplements were tailored to each person's unique results as well. In addition, all participants received personalized lifestyle interventions, such as counseling on exercise, diet, blood pressure control, sleep hygiene and stress reduction.

Everyone in the CEDAR trial has a family history of Alzheimer's, but the majority had no signs of cognitive decline when the study began, Isaacson said. Of the 154 men and women participating in the research, 35 were diagnosed with mild cognitive impairment, or MCI, due to Alzheimer's, but it was not severe enough to "impact their daily lives," he said.

The original study's main findings were published in 2019. People with MCI saw their performance on cognitive tests for memory and thinking skills improve by nearly 5 points when they followed at least 60% of their lifestyle recommendations (on average, at least 12 of the 21 different recommendations) for 18 months.

However, the 2019 study found that people with mild cognitive impairment who followed less than 60% of the suggestions showed no cognitive improvement -- in fact, they continued to decline by 6 points on average.

The cognitively normal patients with a family history of Alzheimer's disease, called the prevention group, were able to get an "equally impressive" cognitive boost of an average of 4.5 points by following at least some of the lifestyle recommendations. It didn't seem to matter if they followed less than 60% of them, Isaacson said.

"The good news from our study is that there were actually cognitive improvements at 18 months in both women and men when compared to the control populations," Isaacson said. "A lot of the drugs that have been studied aim to delay cognitive decline, but it is harder to show improved cognition over time."

Approximately half of the participants in CEDAR carry at least one APOE gene, which may increase the risk of developing Alzheimer's disease. However, the study found no difference in the intervention's cognitive benefits for those with one or two copies of APOE compared with those without the gene, "so that was also reassuring," Isaacson said.

The new study, published Tuesday in the journal of the Prevention of Alzheimer's Disease, took the original 2019 study a step further by analyzing a subset of participants to see whether there was any difference between men and women when it came to how well the lifestyle interventions work.

"Women have very different and unique risk factors than men for dementia," Isaacson said. "Women have a 39% higher risk of dementia if they have fat accumulating around their midsection.

"And the rapid decline in estrogen during the perimenopause transition can actually be one of the most impactful risk factors for developing Alzheimer's pathology in the brain," he said.

In the new analysis, women in the prevention group, who started the trial with no cognitive issues, demonstrated greater improvements than men in two areas: one of two cardiovascular risk scales and in levels of the good cholesterol, HDL, or high-density lipoprotein, which is protective against heart disease.

Women with mild cognitive decline, called the early treatment group, showed greater improvements than men when it came to average blood sugar levels and two cardiovascular risk scales. This female cohort also had more significant improvements in several important cholesterol (or lipid) biomarkers than men in the early treatment group.

For all participants, complying with an additional 10% of the personalized recommendations resulted in an additional 0.9 point improvement for women and 0.41 points of improvement for men on tests of cognition.

How does reducing cardiovascular risk impact future cognition? Because what's good for the heart is good for the brain, experts say.

"Vascular risk factors such as high blood pressure, high cholesterol, high blood sugar or diabetes may not be the cause of Alzheimer's disease, but it can fast-forward Alzheimer's pathology," Isaacson said. "I would prefer to slam on the brakes rather than rev the engine on the path to cognitive decline."

Finding that women were able to reduce their risk even more than men is welcome news, Isaacson said, as it provides a promising area for future study and gives hope that women can tip the battle against Alzheimer's in their favor.

"By treating people in an evidence-based yet safe way, using multiple lifestyle and medical interventions, we've shown that you can really make an impact on brain health," he said.

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PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that oral presentations on LUMEVOQ and GS030 will be featured at the 2021 ASRS and ESGCT meetings in October. Senior management will also attend and present at several investor and industry conferences in the same month.

Chardan 5th Annual Genetic Medicines ConferenceOctober 4-5, 2021 VirtualBernard Gilly, Co-founder & Chief Executive Officer, will present on Monday October 4, 2021, at 8.00 am ET and the management team will host investor meetings during the conference.

A webcast of the fireside chat will be available at https://bit.ly/3zZOGsM for 90 days.

HealthTech Innovation Days (HTID)October 4-5, 2021 VirtualGenSight Biologics management team will host investor meetings during the conference.

2021 Advanced Therapies Congress LIVEOctober 5-6, 2021 ExCel, London (United Kingdom)Magali Taiel, MD, Chief Medical Officer, will present LUMEVOQ on Wednesday October 6, 2021, at 1.30 pm GMT.

2021 OIS @ ASRSOctober 7, 2021 San Antonio, Texas (United States) & VirtualMagali Taiel, MD, Chief Medical Officer, will present on Thursday October 7, 2021, at 9.09 am CDT.

39th Annual Scientific Meeting of the American Society of Retina Specialists (ASRS)October 8-12, 2021 San Antonio, Texas (United States)

Optogenetics in the Clinic: Safety and Efficacy Updates on the Phase I/II Clinical Trial PIONEER will be presented by Joseph Martel, MD, University of Pittsburgh School of Medicine, United States and Investigator in the PIONEER trial.

2021 ARM Cell & Gene Meeting on the MesaOctober 12-14, 2021 Carlsbad, California (United States) & VirtualMagali Taiel, MD, Chief Medical Officer, pre-recorded a Company presentation that will be available during the conference.

28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)October 19-22, 2021 Virtual

The phase III REFLECT trial: Efficacy and safety of bilateral gene therapy for Leber Hereditary Optic Neuropathy (LHON) will be presented by Patrick Yu Wai Man, MD, University of Cambridge, United Kingdom and Investigator in the REFLECT trial.

Partial recovery of visual function in a blind patient after optogenetic therapy for non-syndromic Retinitis Pigmentosa will be presented by Jos-Alain Sahel, MD, University of Pittsburgh School of Medicine, United States and Co-founder of GenSight Biologics.

BIO-Europe 2021October 25-28, 2021 VirtualGenSight Biologics management team will host partnering meetings during the conference.

GenSight Biologics management team will also present at the following conferences:

Mitochondrial Diseases Conference 2021 by Mitocon // October 15-16, virtualAdvancing Gene Therapy 2021 // October 18-20, Boston, USA & virtual

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

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Clinical Data on GenSight Biologics' LUMEVOQ and GS030 Gene Therapies to be Presented at 2021 ASRS, ESGCT and Several Investor and Industry Meetings...

NEW YORK, April 26, 2022 /PRNewswire/ -- The Insight Partners published latest research study on "Synthetic Biology MarketForecast to 2028 - COVID-19 Impact and Global Analysis By Products (Enzymes, Chassis Organisms, Oligonucleotides, and Xeno-Nucleic Acids), Technology (Measurement & Modeling, Cloning & Sequencing, Genome Engineering, Gene Synthesis, Nanotechnology, and Others), and Application (Medical Applications, Industrial Applications, Food and Agriculture, Environmental Applications, and Others)", the global synthetic biology market is expected to grow from $10.54 Billion in 2021 to $37.85 Billion by 2028; it is estimated to grow at a CAGR of 20.0%from 2021 to 2028.

The synthetic biology market growth is driven by the increasing investments in synthetic biology, advancements in biotechnology, and the rising number of start-ups. However, renewed regulations for biotechnology are restricting market growth.

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Report Coverage

Details

Market Size Value in

US$ 10.54 Billion in 2021

Market Size Value by

US$ 37.85 Billion by 2028

Growth rate

CAGR of 20.0% from 2021 to 2028

Forecast Period

2021-2028

Base Year

2021

No. of Pages

217

No. Tables

111

No. of Charts & Figures

84

Historical data available

Yes

Segments covered

Products, Technology, and Application

Regional scope

North America; Europe; Asia Pacific; Latin America; MEA

Country scope

US, UK, Canada, Germany, France, Italy, Australia, Russia, China, Japan, South Korea, Saudi Arabia, Brazil, Argentina

Report coverage

Revenue forecast, company ranking, competitive landscape, growth factors, and trends

Synthetic Biology Market: Competitive Landscape and Key Developments

In August 2021, New England Biolabs announced the release of its newest loop-mediated is sthermal amplification (LAMP) products: the WarmStart Multi-Purpose LAMP/RT-LAMP 2X Master Mix (with UDG) and the WarmStart Fluorescent LAMP/RT-LAMP Kit (with UDG), which combines the master mix and LAMP fluorescent dye in one convenient kit. The new master mix and kit enable researchers to perform rapid, high-throughput detection of targeted segments of DNA or RNA using various amplification detection methods.

In April 2022, Agilent Technologies Inc. announced the expansion of CE-IVD marking in the European Union for its PD-L1 IHC 28-8 pharmDx as an aid in identifying esophageal squamous cell carcinoma patients for treatment with Bristol Myers Squibb's PD-1-targeted immunotherapeutic OPDIVO (nivolumab), in combination with fluoropyrimidine and platinum-based chemotherapy or OPDIVO in combination with YERVOY (ipilimumab). These combined treatments provide new hope for patients diagnosed with these cancers.

In April 2022, Merck KGaA launchedthe first-ever antibody to achieve ACT label designation. ZooMAb antibodies received the lowest Environmental Impact Factor (EIF) scores in the chemicals and reagents category.

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Global synthetic biology market is segmented by region into North America, Europe, Asia Pacific, the Middle East & Africa, and South & Central America. In North America, the U.S. is the largest market for synthetic biology. Asia Pacific is expected to account for the fastest global synthetic biology market growth.. Asia-pacific region will have lucrative growth opportunities during the forecast period due to government initiatives taken in the region. For instance, in November 2017, China launched the first synthetic biology association to support the market's growth. This association promotes academic research and communication in synthetic biology; it further enhances the comprehensive competitiveness of the Shenzhen synthetic biology industry and domestic and foreign influence. While, North America has huge market potential, followed by Europe in the overall synthetic biology market, which is attributed to government funds, investments made by the major companies, conferences held for the awareness regarding synthetic biology, technological advancements in the field of genomics, and higher acceptance by the consumers. However, the MEA and SCAM will also show a relatively lower opportunity in the next few years.

Synthetic biology is an emerging concept that has numerous applications in the chemicals, agriculture, pharmaceuticals, and energy industries. Various start-ups offer gene synthesis products/services for biological processes. For example, Benchling provides CAD tools, GenScript offers gene synthesis, Ginkgo Bioworks assists in organism engineering, Transcripts (a bioinformatics company) offers solutions facilitating cloud labs/automation, and Glowee offers consumer products. Further, 56 synthetic biology businesses raised more than US$ 3 billion in equity financing in the first half of 2020, compared to 65 companies raising US$ 1.9 billion during the same period in 2020. The new generation of biopharma businesses uses synthetic biology to improve cell treatment and gene therapy and support early cancer detection. Some synthetic biology firms with the most funding in the first half of 2020 are listed below.

Table 1. Synthetic Biology Firms with Most Funding in First Half of 2020

Company

Description

Funding

Sana Biotechnology

Biotechnology, Health Care, Life Sciences, and Product Research

US$ 700 million

Impossible Foods

Food & Beverage and Nutrition

US$ 500 million

Moderna Therapeutics

Biotechnology, Genetics, Health Care, Medical, and Pharmaceutical

US$ 483 million

Apeel Sciences

Agriculture, AgTech, Biotechnology, and Food Processing

US$ 250 million

Memphis Meats

Food & Beverage and Nutrition

US$ 161 million

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Synthetic Biology Market: Segmental Overview

The synthetic biology market, based on product, is segmented into oligonucleotides, chassis organisms, enzymes, and xeno-nucleic acid. The oligonucleotides segment is likely to hold the largest share of the market in 2021. Moreover, the same segment is anticipated to register the highest CAGR in the market from 2021-2028. This is mainly because of the rising application of oligonucleotides in genetic testing/ assays and forensic sciences. Based on technology, the market is segmented into, gene synthesis, genome engineering, measurement & modeling, cloning & sequencing, nanotechnology, and others. In 2021, the gene synthesis segment is likely to hold the largest share of the market. However, the genome engineering segment is expected to grow in demand at the fastest CAGR of 20.8% from 2021 to 2028. Based on application, the market is segmented into medical applications, industrial applications, environmental applications, food and agriculture, and others. The medical applications segment is further segmented into drug discovery & therapeutics and pharmaceuticals. In 2021, the medical applications segment is likely to hold the largest share of the market. Moreover, the similar segment is expected to witness growth in its demand at the fastest CAGR of 20.8% from 2021 to 2028.

North America synthetic biology market includes consolidated markets for countries such as the US, Canada, and Mexico. In recent years, all the three countries in the region are witnessing a sequential change in the synthetic biology market. By geography, North America held the largest global synthetic biology market share. This largest share can be attributed to government funds, investments made by the major companies, conferences held for awareness regarding synthetic biology, technological advancements in genomics, and higher consumer acceptance. Synthetic biology is a major tool for biological advances, which helps in providing potential for the development of biological weapons. Security issues can be avoided by regulating the biotechnology industry through policy legislation.

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The various government initiatives and funding offered are becoming great support for the synthetic biology market to grow in the US. For instance, according to a report by National Center for Biotechnology Information (NCBI), the US government offers approximately US$ 220 million every year toward synthetic biology research and development.

Browse Adjoining Reports:

Synthetic Biology Technology MarketForecast to 2028 - COVID-19 Impact and Global Analysis ByType (Gene Synthesis, Genome Engineering, Sequencing, Bioinformatics, Cloning, Site-Directed Mutagenesis, Microfluidics, Nanotechnology); Application (Medical, Industrial, Food and Agriculture, Others) and Geography.

Oligonucleotide Synthesis Market Forecast to 2027 Global Analysis by Product (Synthesized Oligonucleotides, Reagents, and Equipment), Application (Research, Diagnostics, and Therapeutics), and End User (Academic Research Institutes, Pharmaceutical & Biotechnology Companies, Diagnostic Laboratories, and Other End Users).

Gene Synthesis Market to 2025 - Global Analysis and Forecasts By Products & Services (Services, Consumable, Software), Application (Research & Development Activities, Diagnostics, Therapeutics, Others) End Users (Academic & Research Institutes, Biotech & Pharmaceutical Companies, Diagnostic Laboratories, Others) and Geography.

Genomics MarketForecast to 2027 - COVID-19 Impact and Global Analysis by Technology (Sequencing, Microarray, PCR, Nucleic Acid Extraction and Purification, and Others), Product & Service (Instruments/Systems, Consumables, and Services), Application (Diagnostics, Drug Discovery and Development, Precision/Personalized Medicine, Agriculture & Animal Research, and Others) and End User (Research Centers, Hospitals and Clinics, Pharmaceutical & Biotechnology Companies, and Others).

Molecular Biology Enzymes, Kits, and Reagents MarketForecast to 2028 - COVID-19 Impact and Global Analysis By Product (Enzymes and Kits & Reagents), Application (Epigenetics, Sequencing, Synthetic Biology, Polymerase Chain Reaction, and Other), End User (Biotechnological & Pharmaceutical Companies, Hospitals & Diagnostic Centres, and Academic & Research Institutes), and Geography.

Genome Editing Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Technology (CRISPR, TALEN, Antisense, and Others), Application (Cell Line Engineering, Genetic Engineering, Diagnostic Applications, Drug Discovery, and Others), and End User (Pharmaceutical and Biotechnology Companies, Academic and Research Institutes, and Clinical Research Organizations).

Biohacking Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Product (Sensors, Smart Drugs, Strains, Wearables, Others); Application (Synthetic biology, Genetic engineering, Forensic science, Diagnosis and treatment, Drug testing, Others); End User (Pharmaceutical and biotechnological companies, Forensic laboratories, Others) and Geography.

Enzymatic DNA Synthesis Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Product Type (Custom DNA Synthesis, DNA Library Synthesis); Application (Synthetic Biology, Genetic Engineering, Therapeutic Antibodies, Vaccine Design, Others); End User (Academic and Research Institutes, Biotech and Pharmaceutical Companies, Diagnostic Laboratories, Other) and Geography.

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Contact Sameer JoshiE-mail: [emailprotected]Phone: +1-646-491-9876Press Release: https://www.theinsightpartners.com/pr/synthetic-biology-market

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Synthetic Biology Market Size Worth $37.85 Billion, Globally, by 2028 at 20% CAGR - Exclusive Report by The Insight Partners - PR Newswire