Category Archives: Genetic medicine

Newswise Dr. Daniel Geschwind, the Gordon and Virginia MacDonald Distinguished Professor of Human Genetics, Neurology and Psychiatry at UCLA, was awarded the National Academy of Medicines (NAM) 2022 Rhoda and Bernard Sarnat International Prize in Mental Health in recognition of his pioneering research and leadership in autism genetics.

The Sarnat Prize, which includes a medal and a $20,000 prize, is NAMs highest honor related to the brain and mental health. Geschwind, who was elected to the elite panel in 2011, will receive the award at NAMs annual meeting on Oct. 16.

In its award announcement, NAM credited Geschwind with shaping the modern era of research in autism genetics by helping to create and lead the Autism Genetic Resource Exchange (AGRE) with the Cure Autism Now foundation, the first open-access resource for research on autism spectrum disorder (ASD) that was relied on by scientists around the world. The repository of clinical and genetic information, founded in 1997, has led to discoveries in autism genetics, including the role of rare mutations and inherited variation, and created a model for how scientists can work collaboratively to accelerate research in psychiatric genetics.

The NAM award also recognizes Geschwind, the director of the UCLA Center for Autism Research and Treatment, for his laboratorys highly influential research that has shaped our understanding of autism. His work has focused on translating genetic findings to biological understanding broadly across brain disorders. In ASD, this includes his formulation of the disorder as a developmental disconnection syndrome. He pioneered the study of language and social endophenotypes in genetic studies and demonstrated how transcriptomic and epigenetic profiling could be used to define the molecular pathology in ASD brain and other neuropsychiatric disorders. These latter studies have provided a roadmap for understanding the pathophysiology of neuropsychiatric disorders more broadly. Geschwind over the last decade has also sought to boost inclusion of underrepresented groups in autism research, which includes leading the only study of autism genetics in African Americans.

Dr. Geschwinds pioneering work has shown how research can help us meet the challenge of neurologic and psychiatric disorders by identifying how they may be genetically driven, and his creation of AGRE demonstrated the power of community resources to expand these important research efforts, said National Academy of Medicine President Victor J. Dzau. His transformational work including his efforts to make research on ASD more inclusive makes him highly deserving of this recognition.

This award is a testament to the environment at UCLA that has supported my laboratorys efforts all of these years and the students and post-docs that have been involved in driving this work forward, Geschwind said.

The Sarnat Prize, awarded annually since 1992, recognizes individuals or groups that have demonstrated outstanding achievement in improving mental health. The most recent recipient from UCLA was Dr. Kenneth Wells, a professor of psychiatry and biobehavioral sciences at the David Geffen School of Medicine, who in 2018 was honored for his work improving mental health in underserved communities.

The Sarnat Prize is the second major recent award for Geschwind after he received the American Academy of Neurologys top honor for research, the Cotzias Award, in the spring.

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UCLA researcher awarded National Academy of Medicine prize for work on genetics of autism - Newswise

After a two-year pause caused by the pandemic, the NIH project recently stopped in Colorado.

The program is inviting more than a million people from all walks of life to help build what its calling one of the most diverse health databases of its kind and improve medical treatment across diverse groups.

Nevarez agreed to answer survey questions and share medical information, including her DNA from urine and blood. That all goes into the giant research database. Nevarez said shes glad to help.

I think that is a good program. And I think that it's good to do this kind of stuff for everybody, she said.

Nevarez is originally from Mexico. She said that the programs diverse pool will create a powerful tool for researchers.

I think that that's my main purpose to come here, just to show my people, my community. said Nevarez. She leads a diabetes prevention program with Vuela for Health, a health promotion group working with the Latino community. We do have to do this, we do have to participate.

The Hispanic/Latino community represents nearly 20 percent of the U.S. population but only about 5 percent of participants in clinical trials. That same tiny figure is true for Black Americans too, who make up more than 13 percent of the population, according to the program.

Often, people from diverse backgrounds havent been asked to participate or told why its valuable, said Edgar Gil Rico, an All of Us principal investigator with the National Alliance for Hispanic Health. Why is it important for me? Why is it important for the community? he said.

He answers his own question: to help with the development of medicines and treatments that are tailored to each individual. Its whats called precision medicine. He said health is not just a list of different symptoms that you have.

The health of a person is more comprehensive. Where you live, what kinds of activities you do, your family health history, your genetics, all of these play a role in providing you the right treatment for you, he said.

It will improve treatments in the long run, said Jaharri Asten, who came to the bus at the Lowry Family Health Center to provide samples of her DNA.

I know a lot of people, especially minorities, feel like the doctors don't understand their culture, she said. And so having this data will help inform the doctors.

Asten is a counselor in substance abuse and addiction with Denver Health and said better research could help improve treatment based on genetic and cultural differences. So it would definitely be a good idea to have different samples so that medicine could be more specialized for each person, she said.

Asten was excited about potential scientific advancements. I just thought it was really fascinating, she said. Whenever I get the opportunity to participate in research, I do it because you never know who it's gonna help or who could be impacted in the future.

Across the U.S., more than 370,000 participants have completed all the basic enrollment steps, including providing a biosample, either blood, saliva or urine, according to an All of Us press officer.

Offering up your own DNA samples to build that database may be too much, too risky, for some.

But the program says the information is anonymized and the data is protected and secured.

Mayela Picado, a nutritionist from Boulder, said she was comfortable with the tradeoffs.

I know how research works, she said. We are gonna be a number. So my genetics and all my information would be just a number.

Alok Sarwal is a big proponent of this project. He leads a diverse medical clinic with offices in Denver and Aurora, the Colorado Alliance for Health Equity and Practice. It serves 20 ethnicities.

Hes encouraged community members to participate. Not only we are contributing to the database, but we are also now going to be utilizers of the database, said Sarwal, the CEO and co-founder of the clinic. Its mission is to improve the health of the states immigrant communities through culturally and linguistically appropriate prevention, health education, wellness activities, early detection, and self-management of disease.

His colleague, program manager Suegie Park, gave an example of how this information could help. She said theres a popular blood thinner, developed by a pharmaceutical company. Blood thinners are key to stopping blood clots, preventing heart attacks and strokes.

That medication works with white people, research has shown, she said, more than 75 percent of the time. Very effective!

But its less than 50 percent effective for Asians, she said. And a big share of her clinics patients are Pacific Islanders, for whom it doesnt work so well. Its effectiveness with native Hawaiians is only 27 percent.

These kinds of concerns are why the NIH created the project. The agency was well-aware that, oh, we need multi-ethnicity, Park said.

In Colorado, 2,175 participants have completed all the basic steps, including contributing a biosample. Nearly 80 percent identify with a category the program calls underrepresented in biomedical research. This includes racial and ethnic groups, sexual and gender minorities, rural populations, older adults, people with disabilities and those with lower educational attainment and/or income, according to the program.

So far, 17 percent of Colorado program participants identify as Hispanic, according to the program; thats a bit less than their 21.6 percent share of the states population. The numbers for other ethnic groups also trail their share of the Colorado population: 2 percent Black participants, compared to their 4.3 percent portion of the states population, 1 percent Asian (compared with 3.6 percent) and 1 percent American Indian/Alaska Native (compared with 1.7 percent).

The project is also drawing plenty of attention from researchers, like Dr. Nathan Clendenden, an anesthesiologist and professor at the University of Colorado.

He said cardiovascular disease, which includes heart attack and stroke, is the biggest killer. Typically he only studies patients in the hospital. But that is a limited number of patients and often doesnt include everyone in the community, Clendenen said.

I think that's been a blind spot in research is that it's not necessarily representative, he said.

Now Clendenen and his team are exploring a new way to start addressing that problem. Theyve started a new study, about preventing blood clots, using the vast genetic database from the All of Us research program. (The title of the research: Platelet Hyperreactivity as a Therapeutic Target for Reducing Stroke in Older Adults after Transcatheter Aortic Valve Replacement.)

The drugs already exist. So this is really about taking existing tools and matching patients to them, he said.

Clendenen said research done via the database promises to make medicine more precise, ideally personalizing the treatment of individual patients and improving, even saving, lives.

So far, 31 Colorado researchers registered to use the All of Us Researcher Workbench and nearly two dozen research projects have been initiated, according to the program. Those include studies of cardio-metabolic health outcomes, genetic risk factors for thyroid cancer, complications from cataract surgery, and Dr. Clendenens research on platelet reactivity as a risk factor for stroke.

(For more details, search the public Research Projects Directory.)

Another advantage for participants is to learn more about their own DNA. The All of Us program notes on its website it will only show results a participant wants to see.

That information may include genetic ancestry, including where someones family might have lived hundreds of years ago, genetic traits, such as why a person might love or hate cilantro, whether they may have a higher risk for certain health conditions and how ones body might react to certain medications.

Program representatives said you could learn about diseases that you might have, or like if there's any disorders or anything. So, of course, that's interesting, said Asten, who is interested to see what her genetic profile shows. You could see your ancestry and who your most recent ancestors are. So I thought that would be kind of cool too.

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Medical research doesn't serve everyone. This effort hopes to build one of the most diverse health databases ever - Colorado Public Radio

New advances in medical research and technology are helping the field of precision medicine evolve in many ways. One new study highlights the ways genetic testing and profiling can be extended to help diagnose and treat a variety of conditionseven rashes.

For years, diagnosis and treatment of diseases like breast cancer have benefitted from precise genetic profiling. When the same science is applied to skin cells, the result could be a more definitive way to identifyand eventually treatunusual rashes and skin conditions.

The study, published in Science Immunology, was led by a team at the University of California, San Franciscothe same institution that has pioneered an online diagnostic tool called RashX to help dermatology clinicians diagnose traditionally difficult-to-categorize skin rashes with the submission of genetic data from their difficult cases.

RashX uses the same technology outlined in the study. Raymond Cho, MD, PhD, a UCSF dermatologist and geneticist and co-author of the report says traditionally, skin rashes are diagnosed with visual inspection or even a biopsy. Newer technology borrowed in part from the molecular analysis of cancer cells examines the genetic profile of T cells embedded in the skin, he says.

Cho says he and the other researchers on the team found that there are particular molecular profiles that appear to match different skin rashes. Most of these changes are specific to T cells, a major player in a variety of inflammatory conditions.

Inflammatory conditions are behind the largest class of chronic skin diseases, the study notes, adding that single-cell RNA sequencing can help differentiate between these conditions. The study focused on conditions driven by T cell changes like atopic dermatitis, psoriasis vulgaris, lichen planus, and bullous pemphigoid.

While some of these rashes might have been difficult to classify by traditional methods, the research team was able to use genetic signatures to make a more exact diagnosis that was consistent with therapeutic responses. The goal is to integrate data on rash classification from genetic signatures into a visual diagnostic comparison tool for clinicians on RashX.

Although the technology to identify rashes by their genetic signatures is still evolving, Cho says the study showed promising results. In the future, he says there is a possibility that the same technology could be used to guide targeted treatments.

Currently, once a diagnosis is made through visualization or biopsy, Cho says treatment methods range based on classification of the range from mild to more severe, usually starting with topicals and advancing to other therapies. This genetic data, however, could open the door to a shortcut to the most effective treatments for different kinds of rashes.

This really represents something between the biopsy and different drug trials. It gives you a more specific profile, Cho says. I think hopefully were evolving to a place where its not even a two-dimensional spectrum of mild to severe, but multidimensional. Right now, its about trying to identify a rash, but in the future, it might guide treatment.

Reference:

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Precision Medicine Offers Hope for More Targeted Rash Diagnosis and Treatment - Dermatology Times

REDMOND, Ore. (KTVZ) -- The International Association of Fire Fighters has collected critical funds in the community since 1954 one dollar at a time as part of the Fill the Boot program for the Muscular Dystrophy Association.

TheRedmond Fire Fighters Association Local 3650 willbe continuing this long-standing tradition as its members kick off the annual program raising funds to support MDAs vision to accelerate research, advance care, and advocate for the support of MDA families.

Dedicated fire fighters fromthe Redmond Fire Fighters Association Local 3650will hit the streets with boots in hand asking pedestrians, and motorists, to donate to MDA onThursday, Sept. 15 from 9am-2pm, at the intersections of SW 5th and SW 6th Streets at SW Evergreen Ave.

Individuals and local businesses can also support the fire fighters by donating online at:https://filltheboot.donordrive.com/team/11910

The partnership between MDA and IAFF began in 1954 when the IAFF signed a proclamation designating MDA its charity of choice and vowing to continue raising awareness and funds until cures are found. To date, the nearly seven-decade partnership has raised more than $679 million with involvement from over 300,000 fire fighters nationwide. These funds have led in part to over a dozen FDA-approved drugs in as many years for those with neuromuscular disease. Those treatments were created from MDAs vision to open a new field of medicine and push the boundaries of the medical frontier we call genetic medicine.

What the IAFF has done for MDA over the past 68 years is unprecedented, said Donald S. Wood, Ph.D., President and CEO of MDA. With the support from our partners at the IAFF, MDA is doing the impossible in accelerating research, advancing care, and advocating for people living with neuromuscular disease. We have a mission to empower the people we serve to life longer, more independent lives and we will fulfill this mission together, with the IAFF.

About theIAFF

TheInternational Association of Fire Fightersrepresents more than 326,000 professional fire fighters and paramedics who protect more than 85% of the nation's population. More than 3,500 affiliates and their members protect communities in every state in the United States and in Canada.To learn more visitIAFF.organd follow the IAFF atFacebook,Twitter, andInstagram.

AboutMuscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families.MDAs mission is to empower the people we serve to live longer, more independent lives. To learn more visitmda.organd follow MDA onInstagram,Facebook,Twitter,TikTok, andLinkedIn.

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Redmond firefighters to 'Fill the Boot' for Muscular Dystrophy Association - KTVZ

Marissa Leshnov / The Washington Post / Getty Images

When youve lived through two-plus years of a pandemic, it can feel weird to see disease and good news in the same sentence. But here we are, watching a disease decline, with cautious optimism. Two weeks ago, the World Health Organization announced that monkeypox cases in Europe had fallen so fast, the outbreak could be eliminated there. And while the U.S. recently experienced its first monkeypox death, cases here have fallen by 40 percent between the middle and end of August. In other words, its too early to declare victory and dust off our hands, but the situation is generally improving.

This news shows that public health officials and the public itself got some important stuff right in combating this serious illness. But monkeypox is also a reminder that humans will encounter many potentially dangerous new diseases. COVID wasnt the first, or the last. What stops most diseases from becoming pandemics is as much about luck as it is about human intervention.

This spring, many of us braced ourselves for the worst. Monkeypox seemed mysterious, and cases of it were soaring. But a positive outcome was not surprising to the scientists who study the disease. One of the difficulties Ive faced in public communication is trying to get people to understand that none of us who work in public health thought the sky was going to be falling from monkeypox, said Jay Varma, a professor of population health sciences at Weill Cornell Medical College. We were just concerned that a lot of people were going to suffer needlessly because we had a diagnostic test, a drug to treat this and a vaccine to prevent it all stockpiled. Monkeypox was, in other words, a serious disease that needed attention to make sure vulnerable groups were protected, but it was never likely to become the same kind of massive problem as COVID-19.

In August, scientists surveyed more than 800 men who have sex with men, trying to find out how monkeypox and the education campaigns surrounding it had affected their lives. According to results published by the Centers for Disease Control and Prevention, about half of the men made some important changes to their behavior. Of the 824 surveyed, 48 percent reported reducing their overall number of sex partners, 50 percent said they had reduced their one-time sexual encounters and 50 percent said they had reduced sex with people they met on dating apps and in sex clubs. Those voluntary behavioral changes as well as the public health campaigns that inspired them have been particularly crucial to curbing monkeypox, said Varma and Rodney Rohde, a professor of clinical laboratory science at Texas State University.

Thats because other studies have shown that while one-night stands account for only a fraction of sex happening daily among men who have sex with men about 3 percent of daily sexual relationships those interactions are responsible for about half of daily monkeypox transmissions.

Vaccination campaigns have also been important, but the behavioral changes seem to be more widespread in the high-risk community than vaccination has been, Varma said. The original guidance from the CDC has been refreshingly frank and honest and transparent about what are the behaviors that put people at highest risk and what are the ways in which you can minimize your risk, without questioning whether sex is an essential activity to life, he said.

But had the monkeypox outbreak happened just a few years ago, it might not have been on the radar of anyone outside the most affected communities. Dr. Sonja Rasmussen, a Johns Hopkins University professor of genetic medicine who worked at the CDC for 20 years, remembers a former director at the agency often saying that when public health did its job well, we never heard about it.

New diseases are popping up and entering the U.S. all the time, according to Rasmussen and the other experts I spoke with. But SARS-CoV-2 aside, most of them are swiftly and effectively shut down by the hard work of public health. Remember that MERS outbreak when there were two cases in the U.S.? she asked, referring to the time in May 2014 when a particularly deadly cousin of COVID cropped up in unlinked cases in Indiana and Florida. People would say, I dont even remember that. And thats because we dealt with it.

Were more likely to hear about these diseases now because everyone is much more primed to pay attention after a couple of years of COVID. But the reality is that thousands of people nationwide are working to ensure those diseases dont spread unnoticed, that the highest-risk populations are treated, and that we dont end up constantly marinating in preventable pandemics. Thats the good news.

The bad news: Not every pandemic is a preventable one. We did get a little lucky [with monkeypox], Rohde said. Yes, theres pain involved and some risk of death, but if and when this disease is nipped in the bud, that will be in part because the virus makes itself relatively easy to prune. Its not a respiratory virus that people can easily spread to strangers at the bus stop. The mode of transmission, primarily through sex, limits who can spread to whom. The transmission rate is also different from that of COVID, he said. And the mode of transmission means the virus affects primarily a high-risk group rather than all of society, so its easier to change behavior and administer pharmaceutical treatments. Monkeypox is also a DNA virus, not an RNA virus like SARS-CoV-2, so it mutates less than COVID and can be prevented with older, existing vaccines. Those are the kinds of outbreaks humans can stop from turning into pandemics. Of course, both scientists and the public have to take action when they pop up, but its relatively easy to manage.

Most new or new-to-us diseases that appear will have more in common with monkeypox than with COVID. Theyll be dealt with. And youll forget you ever saw them on the news. But, eventually, another pathogen will come along thats more challenging just by its nature another fast-spreading, fast-mutating respiratory virus that hits everybody all at once. I am concerned as we move away from COVID that were going to say, Thats our pandemic. We dont need to fund [public health infrastructure] anymore, Rasmussen said.

Unfortunately, one of the biggest takeaways from this monkeypox outbreak and how it was handled is a paradox. You dont need to assume that every new disease you hear about will be another uncontrollable pandemic, so you can let that tension go. But, at the same time, that doesnt mean another pandemic wont happen in your lifetime. Somebody needs to be on the job, paying attention.

It doesnt matter if youre tired, if youre fatigued, if youre done with it, Rohde said. Those [infectious diseases] dont care. They never get tired.

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Why Monkeypox Wasnt Another COVID-19 - FiveThirtyEight

MANHASSET, N.Y.--(BUSINESS WIRE)--Since 2016, scientists at The Feinstein Institutes for Medical Research have studied the genetic and cellular makeup of menstrual blood or menstrual effluent (ME) to diagnose endometriosis. Today they published a new study in Springer Natures BMC Medicine that shows their ability to use ME to identify patients who may have endometriosis.

Endometriosis occurs when uterine-like tissues grow outside of the uterus and form lesions. The condition affects one in 10 reproductive-age females, resulting in chronic, often debilitating pain or infertility and other medical complications. Due to the lack of non-surgical diagnostic tools, it can often take seven to 10 years to be diagnosed with endometriosis. Currently, invasive laparoscopic surgery is the only definitive diagnostic method.

The research, led by Peter Gregersen, MD, and Christine Metz, PhD, analyzed the genetic and cellular differences in healthy controls versus endometriosis subjects to find common biomarkers that could lead to new diagnostic approaches and potential treatments. Building off more than seven years of research, the new study published in BMC Medicine outlines the first use of single-cell RNA-sequencing (scRNA-Seq) to compare endometrial tissues in freshly collected ME from 33 study participants.

Millions of adolescents and women suffer from endometriosis without a proper diagnosis, delaying their care and extending their pain, said Dr. Metz, professor in the Institute of Molecular Medicine at the Feinstein Institutes and co-director of Research OutSmarts Endometriosis (ROSE) study. This new paper describes the potential for a novel screening tool to identify endometriosis earlier and enable patients to get the help they need.

The published study shows for the first time that the characteristics of endometrial tissue that shed into ME are distinct in patients with endometriosis compared to control (healthy) subjects. When combined with clinical symptoms, there is a potential to use ME to screen or diagnose adolescents and women who may be suffering with symptoms of endometriosis.

ROSE study research helps us understand the molecular and genetic makeup of endometrial tissues in ME from women with endometriosis, said Dr. Gregersen, professor in the Institute of Molecular Medicine at the Feinstein Institutes and co-director of the ROSE. More than 2,000 women have participated in the ROSE study to date and we are grateful to them for helping us to produce knowledge that will improve patients lives.

In order to further validate these findings, the team has recently initiated a new clinical trial to compare ME from symptomatic women who have not been diagnosed but will undergo the necessary surgery as part of their standard care to determine if they have the condition. Studies are also underway to investigate ME in symptomatic and non-symptomatic adolescents to predict endometriosis at earlier ages and stages.

While endometriosis is a common condition, there continues to be a lack of diagnosis and proper early intervention, said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes. These important findings by Drs. Gregersen and Metz hold promise to change our understanding of this disease and focus on improving the diagnosis and care they need.

About the Feinstein Institutes

The Feinstein Institutes for Medical Research is the home of the research institutes of Northwell Health, the largest health care provider and private employer in New York State. Encompassing 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health system science, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine a new field of science that has the potential to revolutionize medicine. For more information about how we produce knowledge to cure disease, visit http://feinstein.northwell.edu and follow us on LinkedIn.

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Endometriosis Researchers One Step Closer to Diagnosing Condition With Menstrual Blood - Business Wire

The relatively short history of cell and gene therapy is not lacking in dramatic moments. A previous outlier, this vibrant field now represents the next great hope and so, when roadblocks to progress are removed or even lowered, theres reason to celebrate. Here, seven members of The Medicine Maker Power List 2022, reflect on the most impactful cell and gene milestones.

There have been many significant breakthroughs in cell and gene therapy over the past few years. Specifically in gene-modified cell therapy, the CAR T story is remarkable. Over the past several years, multiple autologous CAR T therapies have been successfully translated from bench to bedside and received marketing authorization as potentially curative therapies for patients with recalcitrant cancer indications: Kymriah and Yescarta for treating r/r/ ALL, MCL, and LBCL, and Abecma for treating r/r multiple myeloma.

Equally impressive in gene therapy, Zolgensma, an AAVSMN1 gene replacement product, has been developed for use as a one-time gene replacement treatment for infants with spinal muscular atrophy (SMA). The 15 year follow-up study these tiny patients are enrolled in after treatment will inform us on the long-term safety and efficacy of gene replacement therapy.

These products have been translated by academia and SMEs and partnered for advanced development with pharma to achieve both medical and commercial success.

The biggest breakthrough is our increasing ability to edit genes with a growing number of new classes of gene editing tools. This advance has led to the boom of CAR T products and is opening the path to cell engineering and in vivo gene therapy.

In parallel, we are seeing an evolution from viral delivery to alternatives with growing payload capacity. This will, as we are already seeing, lead to cures in diseases where that was unthinkable before!

Cell and gene therapies are at the forefront of innovation and transforming how we treat and potentially cure certain diseases. Cell and gene therapies(CGTs) have the potential to treat severe diseases, such as cancer, as well as rare diseases. Several such therapies are now on the market, including a treatment for an inherited retinal disease that causes blindness. That particular CGT represents an important medical milestone because it was the first curative gene therapy approved for use. Personally, I was excited and humbled at the same time to have been the Global Head bringing this transformational therapy to patients around the world. Many other CGTs are now in development and hopefully will lead to an expansion of the still-limited treatment options available to many patients and transform the clinical paradigm.

An important breakthrough? The demonstration that gain-of-function genetically weaponized somatic cells are potent pharmaceuticals in their own right: living synthetic therapeutics (LSTs).Case in point, after a quarter century of work with TILs and LAKs struggling to meet utilitarian endpoints, enter gain-of-function CAR engineering, and thus history is made.The same paradigm of cell gain-of-function genetic enhancement can readily be applied to alternate somatic cell platforms think MSCs and iPSCs with a limitless potential to improve clinical outcomes for acute and chronic ailments.

Id like to emphasize three milestones. First, the commercialization of gene therapies in general. The efficacy and safety have improved a lot since the 1990s.

Secondly, the explosion of immunotherapies. Onco-hematology has become a major opportunity for patients with otherwise lethal blood cancers.

Finally, the advances in gene editing technologies. These have opened the door to new therapies which we would have considered utterly incredible a few years ago.

The recent approval for Yescarta in second-line (2L) relapsed/refractory large B-cell lymphoma (LBCL) means that an order-of-magnitude more patients just became eligible for potentially curative therapies. One recent industry insight from Celltelligence suggested that moving from 3L to 2L will potentially double the targetable population in diffuse LBCL alone for CAR T cell therapy. As cell therapies move up the treatment paradigm and cell-based therapeutics are eventually approved to treat a range of cancers, the spotlight will turn (again) to manufacturing capacity. At Cellares, our belief is that high-throughput, end-to-end automation is set to revolutionize cell therapy manufacturing, allowing us to deliver more doses at lower cost to meet the demand. Its a truly exciting time for our industry!

The success of the CAR T cell therapy approach and how it has led to cures for childhood leukemias and lymphomas is an amazing story. Thanks to these incredible advances, kids who would no longer be here today are now effectively cured, and are going to live long, relatively healthy lives without suffering the long-term side effects of traditional chemotherapy and radiation. By allowing investigators to be highly creative in developing this approach, a fascinating new treatment process was developed, for both autologous and allogeneic CAR T cell therapies. Now, an entire industry has been born from utilizing patients and donors stem cells and a modified version of the AIDS virus to cure leukemia. This is truly a mind-blowing advancement that combines so many complex processes and biologics and really showcases the power of creative investigators to come up with amazing new treatment solutions.

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The Biggest CGT Breakthroughs Through the Eyes of Our 2022 Power List - The Medicine Maker

Krzysztof J. Misiukiewicz, MD, discusses when targeted therapy may be used for patients with NTRK-positive thyroid cancer.

Krzysztof J. Misiukiewicz, MD, associate professor of hematology and medical oncology at the Icahn School of Medicine at Mount Sinai and clinical director of the Center for Personalized Cancer Therapeutics at the Tisch Cancer Institute, discusses when targeted therapy may be used for patients with NTRK-positive thyroid cancer.

Misiukiewicz says that patients with thyroid cancer will first receive surgery and/or radioactive iodine therapy. Those who continue to progress following total thyroidectomy and receiving radioactive iodine need further treatment. Genetic testing can reveal potential targets for therapy including RET, BRAF V600E, and NTRK, which have different recommended regimens.

Those who have an NTRK fusion, are iodine-refractory, and have no alternative treatment options are eligible for the NTRK inhibitors larotrectinib (Vitrakvi) and entrectinib (Rozlytrek). These therapies are approved for patients with any solid tumor with an NTRK fusion, which can also include salivary gland tumors and soft tissue sarcoma.

According to Misiukiewicz, larotrectinib would be his preferred next line of therapy for any patient with iodine-refractory thyroid cancer with an NTRK fusion based on the efficacy seen with NTRK inhibition.

TRANSCRIPTION:

0:08 | In thyroid cancer, we treat patients [who] are iodine-refractory, meaning that they underwent total thyroidectomy; they underwent treatment with radioactive iodine and for any reason, those treatments were not effective, and the cancer is still progressing, so we label those patients as iodine-refractory patients. In those situations, those patients are being referred to me, where I do first genetic testing. And then I would consider treating those patients with larotrectinib. So they have to have evidence of progressive cancer that hasn't responded to previous treatments such as surgery or radioactive iodine.

Whenever I have [patients with] the NTRK-positive disease, I prefer targeted therapy. Currently we have 2 drugs on the market that are approved; my preferred drug is larotrectinib. That is approved for NTRK-mutated patients. And this is the drug that usually I choose when I have patients with the NTRK-positive mutation.

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When to Use Targeted Therapy for Iodine-Refractory Thyroid Cancer - Targeted Oncology

When the first humans moved out of Africa, they carried their gut microbes with them. Turns out, these microbes also evolved along with them.

The human gut microbiome is made up of hundreds to thousands of species of bacteria and archaea. Within a given species of microbe, different strains carry different genes that can affect your health and the diseases youre susceptible to.

There is pronounced variation in the microbial composition and diversity of the gut microbiome between people living in different countries around the world. Although researchers are starting to understand what factors affect microbiome composition, such as diet, there is still limited understanding on why different groups have different strains of the same species of microbes in their guts.

We are researchers who study microbial evolution and microbiomes. Our recently published study found that not only did microbes diversify with their early modern human hosts as they traveled across the globe, they followed human evolution by restricting themselves to life in the gut.

We hypothesized that as humans fanned out across the globe and diversified genetically, so did the microbial species in their guts. In other words, gut microbes and their human hosts codiversified and evolved together just as human beings diversified so that people in Asia look different from people in Europe, so too did their microbiomes.

To assess this, we needed to pair human genome and microbiome data from people around the world. However, data sets that provided both the microbiome data and genome information for individuals were limited when we started this study. Most publicly available data was from North America and Western Europe, and we needed data that was more representative of populations around the world.

So our research team used existing data from Cameroon, South Korea and the United Kingdom, and additionally recruited mothers and their young children in Gabon, Vietnam and Germany. We collected saliva samples from the adults to ascertain their genotype, or genetic characteristics, and fecal samples to sequence the genomes of their gut microbes.

For our analysis, we used data from 839 adults and 386 children. To assess the evolutionary histories of humans and gut microbes, we created phylogenetic trees for each person and as well as for 59 strains of the most commonly shared microbial species.

When we compared the human trees to the microbial trees, we discovered a gradient of how well they matched. Some bacterial trees didnt match the human trees at all, while some matched very well, indicating that these species codiversified with humans. Some microbial species, in fact, have been along for the evolutionary ride for over hundreds of thousands of years.

We also found that microbes that evolved in tandem with people have a unique set of genes and traits compared with microbes that had not codiversified with people. Microbes that partnered up with humans have smaller genomes and greater oxygen and temperature sensitivity, mostly unable to tolerate conditions below human body temperature.

In contrast, gut microbes with weaker ties to human evolution have traits and genes characteristic of free-living bacteria in the external environment. This finding suggests that codiversified microbes are very much dependent on the environmental conditions of the human body and must be transmitted quickly from one person to the next, either passed down generationally or between people living in the same communities.

Confirming this mode of transmission, we found that mothers and their children had the same strains of microbes in their guts. Microbes that were not codiversified, in contrast, were more likely to survive well outside of the body and may be transmitted more widely through water and soil.

Our discovery that gut microbes evolved right along with their human hosts offers another way to view the human gut microbiome. Gut microbes have passed between people over hundreds to thousands of generations, such that as humans changed, so did their gut microbes. As a result, some gut microbes behave as though they are part of the human genome: They are packages of genes that are passed between generations and shared by related individuals.

Personalized medicine and genetic testing are starting to make treatments more specific and effective for the individual. Knowing which microbes have had long-term partnerships with people may help researchers develop microbiome-based treatments specific to each population. Clinicians are already using locally sourced probiotics derived from the gut microbes of community members to treat malnutrition.

Our findings also help scientists better understand how microbes transition ecologically and evolutionarily from free-living in the environment to dependent on the conditions of the human gut. Codiversified microbes have traits and genes reminiscent of bacterial symbionts that live inside insect hosts. These shared features suggest that other animal hosts may also have gut microbes that codiversified with them over evolution.

Paying special attention to the microbes that share human evolutionary history can help improve understanding of the role they play in human well-being.

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Humans evolved with their microbiomes like genes, your gut microbes pass from one generation to the next - The Conversation

SAN DIEGO, Calif., and SUZHOU andSHANGHAI, China, Sept. 14, 2022 /PRNewswire/ -- Gracell Biotechnologies Inc. (NASDAQ: GRCL) ("Gracell"), a global clinical-stage biopharmaceutical company dedicated to discovering and developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced that the management team will participate in and attend one-on-one meetings at three investor conferences in September 2022 as follows:

Citi Hong Kong China Corporate Day VirtualOne-on-one meetings: Tuesday, September 20, 2022 Wednesday, September 21, 2022

Cantor Oncology, Hematology & HemeOnc ConferencePanel Presentation: Wednesday, September 28 at 4:15 p.m. ETOne-on-one meetings: Wednesday, September 28, 2022Location: New York, NY

Jefferies Cell & Genetic Medicine SummitFireside Chat: Friday, September 30 at 2:00 p.m. ETOne-on-one meetings: Friday, September 30, 2022Location: New York, NY

A webcast of the fireside chat will be available on the News and Events section of Gracell's investor website. A replay of the webcast will be available for 30 days following the event.

About Gracell

Gracell Biotechnologies Inc. ("Gracell") is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms and SMART CARTTM technology module, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal cell quality, high therapy cost and lack of effective CAR-T therapies for solid tumors. For more information on Gracell, please visit http://www.gracellbio.com and follow @GracellBio on LinkedIn.

Media contactMarvin Tang[emailprotected]

Investor contactGracie Tong[emailprotected]

SOURCE Gracell Biotechnologies Inc.

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Gracell Biotechnologies to Participate in Three Upcoming Investor Conferences - PR Newswire