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Spotlight: Women in Science: Dr. Judith Kassis on Pursuing the Unexpected – National Institute of Child Health and Human Development

Posted: September 8, 2022 at 2:03 am

Dr. Kassis with members of her laboratory.Credit: NICHD

During a research career that spans four decades, NICHDs Judith Kassis, Ph.D., has learned many lessons along the way. For the last 23 years, she has headed the Section on Gene Expression at NICHD, and before that, she spent 12 years as a researcher and regulator at the Food and Drug Administration (FDA). Dr. Kassis is a leader in the field of transcriptional gene repression by Polycomb group proteins (PcG), and her laboratory explores how genes are regulated during embryonic development.

I like to follow up on unexpected results. Be open-minded about the answer, and test the hypothesis. A lot of experiments today are designed to prove a hypothesis, when you should always evaluate your idea and assumptions first, shared Dr. Kassis. Todays research environment limits ones ability to do this, but early on, I could chase whatever I wanted in the lab. When I started my graduate studies, I didnt even think about my career. I simply loved science and wanted to do it.

Childhood photo of Dr. Kassis (left) at age 1.5 with her sister Diane, age 3.Credit: Judith Kassis, Ph.D.

Dr. Kassis was the second of six children, raised in Sacramento, CA. Her paternal grandparents were Lebanese American and had an arranged marriage. They lived in North Dakota, where her grandfather owned a candy store, but the Dust Bowl forced the couple to relocate with extended family in California. Dr. Kassis father worked as a grocery store manager and insurance salesman. He later went back to school to become a certified financial planner. Dr. Kassis mother, who grew up in Minneapolis, was a college graduate and medical technician. She became a full-time homemaker soon after becoming a mother.

Looking back, Dr. Kassis is proud of carving out her career and having the opportunity to conduct research at NIH. My mom didnt work outside of the home, and I didnt know what I was going to do. Its amazing to consider where I ended up with this career when I didnt have a plan, she said.

Dr. Kassis mother was active in the American Field Service , which was known at the time for its international student exchange programs. They also had programs to learn about Native cultures, explained Dr. Kassis. When she was a senior in high school, she was an exchange student in Kotzebue, Alaska, about 30 miles north of the Arctic Circle, and spent a semester at a Bureau of Indian Affairs school. I was there during the fall and got to see the Northern lights. What I remember most is looking at the stars over the the Kotzebue Sound and the tundra, so beautiful. I was so lucky to be able to experience such a different environment and culture.

Her parents also planned independent trips for their children. When I was 18 years old, my older sister and I took Greyhound buses all over the country and visited friends of my parents. Remember, we didnt have cell phones back then, she said. Dr. Kassis and a younger brother also visited Mexico, where they took buses to Guatemala and El Salvador. My parents were very loving, generous people, who encouraged us to be curious and try different things.

For college, Dr. Kassis attended the University of California at Santa Barbara, initially with no idea of what she wanted to study. This is going to sound extremely nerdy, but I took a biochemistry class and loved it. I studied so hard that I could actually see the connections between all the different biochemical pathways, she said. For my final exam, I got an A++, and my professor helped me apply for a summer research fellowship through the National Science Foundation. It was the summer of 77, and I absolutely loved working in the lab. For her fellowship, Dr. Kassis studied malaria and enzymatic pathways at the University of California, Riverside, and then published her first paper.

During her senior year, Dr. Kassis worked in the laboratory of Leslie Wilson, Ph.D. Research was my passion, and I realized there was nothing else I wanted to do. Thats why I applied for graduate school, she explained. After graduating with highest honors and a degree in biochemistry and molecular biology, Dr. Kassis moved in 1978 to the University of Wisconsin, Madison, for her doctoral studies. I knew I wanted to move out of state, and Wisconsin had one of the best biochemistry programs in the country, she said.

Her thesis advisor was Jack Gorski, Ph.D. , one of the discoverers of the estrogen receptor. He was very smart and kind. I remember I had the choice of three topics and, after I picked one, I had to learn how to be independentthat was his philosophy. I had to think for myself, figure out who to talk to, and learn how to seek help, she said. It was a nice lab, and there were a lot of women. Overall, it was a very good experience. During those years, Dr. Kassis studied estrogen receptor recycling and met her future husband, Scott Stibitz, a fellow graduate student.

While finishing graduate school in 1983, Dr. Kassis decided to change fields. She found a new postdoctoral position in a lab headed by Patrick O'Farrell, Ph.D. , at the University of California, San Francisco, where she studied evolutionary conservation of the Drosophila engrailed gene, an important developmental gene in fruit flies that has counterparts in other living organisms, including people. It was there that she also developed a lifelong interest in how developmental genes are regulated by DNA sequences that are far away (i.e., tens to hundreds of kilobases) from where the activity begins at a transcription unit. Eventually, she would focus on the field of developmental epigenetics.

In my opinion, it was the best time to be in biology. The homeodomain was discoveredflies had it, humans had it. For the first time, people recognized that animals had so much in common at a genetic level. It was very exciting, she said. But at that time, cloning a gene and sequencing it was a whole project. For my postdoc, I cloned the engrailed gene from a related Drosophila species and sequenced it. Experiments have completely changed now. Its great that you can do more, but back then, you had more time to think.

Dr. Kassis published several postdoctoral papers and got married as she wrapped up her four-year position. I had great advisors, but there was no career mentoring back then. People didnt really talk much about careers. The biotech industry was just starting. No one asked me, Do you want to be a [principal investigator]? Everything was just about the science, she explained. Her husband received a job offer from the FDAs Center for Biologics Evaluation and Research (CBER). So, Dr. Kassis applied for jobs in the Washington, DC, area and received a job offer from CBER, too.

In 1987, she and her husband moved to the East coast, where Dr. Kassis split her time between leading a small research laboratory and regulating products made in living cells for CBER. At the time, the FDA had laboratories on the NIH campus in Bethesda, MD, and Dr. Kassis collaborated often with NIH researchers. Like all principal investigators at CBER, her progress was reviewed under NIHs tenure system, and she received tenure in 1994. The next few years brought many exciting changes.

Dr. Kassis was featured in the NIH Catalyst when she received tenure in 1994.Credit: Judith Kassis, Ph.D.

The first was related to Polycomb Response Elements (PREs). During embryonic development, some genes must be silenced or inactivated at certain times and places, for instance, in the development of different tissues and organs. This silencing is orchestrated by Polycomb group proteins (PcG), which must be recruited to the gene in question. The genes themselves contain special sequences called PREs, which bring in an intermediaryPRE DNA-binding proteinsto recruit the PcGs.

Dr. Kassis and colleagues made a string of discoveries beginning with an unexpected observation in fruit flies that enabled Dr. Kassis to easily identify PREs. She initially wanted to identify regulatory DNA by cloning it and placing it into a vector to make transgenic flies. The vector also contained a marker for eye color, a common research practice to easily identify transgenic flies from normal flies. When white-eyed flies were injected with the vector, their transgenic offspring had colored eyes. Normally, flies with two copies of the transgene (i.e., homozygotes) have a darker eye color than those that only have one copy (i.e., heterozygotes).

However, in Dr. Kassis experiments, when certain fragments of DNA were cloned into the transgene vector, the eye color of the homozygotes was lighter than that of heterozygotes. In fact, most of these homozygotes had white eyes, suggesting that the eye color marker was somehow blocked or silenced. Dr. Kassis called this phenomenon pairing-sensitive silencing. Later, she discovered that this phenomenon was caused by the PREs that she had cloned into the vector. When I presented my findings at a conference, people were very excited, and some even mentioned they had similar observations but had not followed up. I think thats how I distinguished myselfI follow up on unexpected, interesting findings, she said.

Dr. Kassis and her laboratory also discovered the first PRE binding protein, Pho, and later identified three more: Pho-like, Spps, and Combgap. The benefit of having two jobs at FDA was that I didnt have to worry about publishing in high-profile journals. I didnt have to be a bigshot. I could just do good work, explained Dr. Kassis. At that point, I was just doing what I was interested in, and I found [PREs], and it was so much fun.

Dr. Kassis with her daughter Sandy in the 1990s.Credit. Judith Kassis, Ph.D.

Dr. Kassis worked at CBER for 12 years, heading two licensing committees and her small lab. During that time, she also had her two children. It was very hard to do both the regulatory work and the research. I could see that I was going to have to make a decision, especially once you have kids. Thats like three jobs: the lab, the regulatory work, and your children, she said. She successfully applied for an opening at NICHD in 1999 to head a research lab, and she brought along her staff scientist, Lesley Brown, Ph.D., and a postbaccalaureate fellow. It was a very good time to look for a new job, and I want to highlight Lesleys contribution. Shes a transcription factor expert and discovered Pho in my lab. Im not sure I would have gotten the [NICHD] job without her and the timely publications.

NICHD colleague Paul Love, M.D., Ph.D., said that Dr. Kassis is patient, inquisitive, supportive, and always willing to listen. Scientifically, she exemplifies the model investigator. Early in her career, she identified an important but very complex question in biological science, Polycomb-mediated gene repression, and she has devoted her career to steadily chipping away at the problem. Along the way, she has come up with new insights and challenged existing paradigms. It doesnt get any better than that!

When Dr. Kassis first started her lab, she did a lot of work at the bench. Even today, you can find her observing fly embryos under the microscope and doing her own immunostaining. On some of her early papers, she was the only author because the lab was so small. It can be difficult hiring good postdocs when youre starting out, said Dr. Kassis. My advice for tenure-track investigators is to attend meetings, talk to people, talk to journal editors, and promote yourself. If youre shy, pretend you are not.

She also recommends finding a core scientific family. Dr. Kassis attends a small, regional meeting, which originally consisted of five laboratories interested in somatic chromosomal pairing in flies. We collaborate and review each others papers before submission. I also found a postdoc through this group. If youre not currently part of one, consider starting your own group.

At one point, Dr. Kassis considered taking a break from research to teach children, like her son, Zack, who have autism spectrum disorder (ASD). When he was younger, one of the hardest things we did was fight for resources. It took a lot of time and emotional energy, but things have gotten better for children and their families. Dr. Kassis also appreciates the flexibility of science that can enable a work-life balance. If I had to take my son to therapy, I could go and make up the time later. But I didnt travel to meetings as much as I might have. My husband was very supportive, so I could have gone, but I didnt want to leave them. After graduating from high school, Zack attended a program in Arizona to learn to live independently. He still resides there, with limited assistance from an agency that supports adults with ASD.

Dr. Kassis (center) on vacation with her son Zack (left) and husband Scott (right).Credit: Judith Kassis, Ph.D.

Many colleagues have benefitted from Dr. Kassis career and mentorship. Todd Macfarlan, Ph.D., recalled his early years as a tenure-track investigator at NICHD, when the intramural program reorganized into affinity groups that included several labs. Dr. Kassis became the head of our affinity group, and she demonstrated tremendous leadership, creating a fun and exciting environment for sharing our science with our close colleagues. This was a truly exceptional incubator for me, personally, and a major part of my labs early success, he said.

Mitzi Kuroda, Ph.D. , a professor at Harvard Medical School who also studies Polycomb proteins, co-authored a review with Dr. Kassis. She shared, Dr. Kassis discoveries include the foundational analysis of Pho, as well as recent elegant genetic studies on the establishment of repressive Polycomb domains and their boundaries. Her scientific acumen and creativity are especially evident in her truly unexpected discoveries of pairing-sensitive silencing and P-element homing. For these important scientific insights, as well as for her leadership and generosity, she has been irreplaceable as a mentor and colleague.

Dr. Kassis, who is now in her mid-60s, plans to retire next year. Her timeline for retirement has been influenced by various decisions and experiences over the last several years. One of the most pivotal was the unexpected death of her older sister Diane, who passed away four years ago from an aggressive cancer. We were very close and had shared a room growing up. Her death made me realize that theres more I want to do. I want to spend more time with loved ones, said Dr. Kassis. In addition to her son, she has a daughter, Sandy, who lives in Louisiana, and she enjoys visiting both of them. Dr. Kassis and her husband also raise chickens and bees and have three dogs. While I never planned on working until my 80s, I realize now that things can happen.

About eight years ago, Dr. Kassis considered switching fields but never did. Instead, she decided to tackle the unfinished projects that accumulated over the years. She also stopped hiring postdoctoral fellows once she committed to her retirement timeline. These decisions resulted in a perfect pairing of projects ideal for postbaccalaureate fellows, who generally stay for one or two years before moving on to graduate school. Dr. Kassis also does not have to concern herself with high-profile journals, which are more important for a postdoctoral fellows future job prospects.

Anna Horacek, a former postbaccalaureate trainee currently in the Molecular Cell and Biology Program at the University of California, Berkeley, is grateful for her time in Dr. Kassis lab. Notably, Dr. Kassis recognizes that each mentee is unique. While I struggled with communication and specific organizational skills, others needed support with critical thinking and designing experiments. With these objectives in mind, Dr. Kassis altered her approach for each person. She encouraged us to think of future experiments, set up one-on-one meetings to discuss professional development, and provided opportunities for us to present our research.

Currently, one of the labs most exciting projects looks at the stability of a genes on and off transcriptional state and how it is regulated. The lab is using a 79-kilobase transgene to study two epigenetically regulated enhancers, regulatory DNA sequences, important in the Drosophila wing. She and her colleagues found that deletion of either of these enhancers from the 79-kilobase transgene causes flies to have mutant wings. Surprisingly, deletion of the same enhancers from the endogenous locus (i.e., where it occurs naturally in the flys genome) does not cause the same wing mutations.

No one has moved a domain and then tried to fix it. Thats essentially what were doing, said Dr. Kassis. The only difference between the transgene and the endogenous locus is that the endogenous locus has boundaries that stop the spread of epigenetic marks and enhancer activity. These boundaries make it so that everything is concentrated inward in the gene, she explained. The lab recently found that adding these boundaries to their transgene also influenced the genes on and off transcriptional states, which fits into Dr. Kassis hypothesis. Dr. Kassis aims to wrap up this project and finish writing six papers by next year.

What Ive learned is how redundant and resilient development is, said Dr. Kassis. When you have a developmental disorder, the range of phenotype is wide because theres backup in the system. Im fascinated that even though you can make so many mutations in a gene, there are just as many redundant enhancers that enable the fly to live. But how many subtle defects do they have? Its all very interesting.

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Spotlight: Women in Science: Dr. Judith Kassis on Pursuing the Unexpected - National Institute of Child Health and Human Development

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10 wellness retreat trends to know about – Cond Nast Traveller

Posted: September 8, 2022 at 2:03 am

1. All-out lagoons

Sure, its cold outside in Iceland, but bubbling below the surface, geothermal energy is turning up the heat on wild-water dips in the land of fire and ice. Since the 12th century, Icelanders have soaked in silica-rich pools and hidden hot springs. But a new generation of super-spas is delivering greater comfort without making the experience any less rugged. By the same architects that created Keflaviks Blue Lagoon and the recent Geosea baths, Forest Lagoon, ensconced in rock and wood, is a timber-clad complex that seems to have emerged out of the sap-scented deep. Overlooking Valaskogur forests birch and pine, the Eyja fjord and the city of Akureyri, it includes a restaurant, two infinity pools, two swim-up bars, a cold tub and sauna. An hours drive north-east in the town of Husavik, Geoseas cliff-side seawater pools overlook a bay visited by humpbacks; while further east, near Egilsstair, Vok Baths have two pools suspended in a lake with water clean enough to drink. Down south, Reykjaviks Sky Lagoon has mastered the holistic hot-spring experience. A seven-step ritual includes a circuit of sauna, rain room and body scrub, waterfall shower and drinks at a swim-up bar: good vibes all round. Sarah Marshall

At the new Chenot Molecular Lab at Chenot Palace Weggis in Switzerland, epigenetics has gone up a notch. New mRNA-based technology at the brands sleek flagship on Lake Lucerne analyses gene activity, determines biological aging and prescribes treatment via supplements, nutrition, stress control and lifestyle changes. Inflammation, oxidative stress, hormonal imbalance and the structural integrity of tissue are highlighted. Its a modern approach, says Dr George Gaitanos, Chenots chief scientist and COO, where health is defined as what is unseen. Plus, its not all doom and gloom, because the epigenetic picture is dynamic and totally malleable, he promises. DNA is static, the 21,000 piano keys you were born with. But then you play the piano and create the melody. Are you hitting good notes, or are you too aggressive? The idea is to uncover the causes of aging and health issues years before the onset of decline. Lydia Bell

Healing Holidays can arrange a seven-night Advanced Detox programme from 6,449 per person full board; healingholidays.com. The Chenot Molecular Lab costs an extra 1,050

Sophie Delaporte / Trunk Archive

The meditation of the future is about diving deeper, a plunge into existential fathoms rather than a fey skip through the shallows of mindfulness. Mandali, a seductively minimalist new retreat in Italy, slides a side order of sybaritic comfort alongside pristine meditation instruction, with stunning views over Lake Orta inspiring guests into awed silence. At Eremito, Umbrias monastery-meets-eco hotel, meditators sleep in celluzze, or hermit cells, and the aura of peace here is palpable. Zenways life-shifting and intensive three-day retreats (in the UK, mainland Europe and the USA) are definitely not for the faint-minded. The schedule runs from dawn to nudging midnight, with 13 sessions every day. As you sit in pairs, it kicks off with a simple question: Tell me who you are? The idea is that the ego plays Twister with itself before finally giving in and settling into Zen awakening, or satori. It works and its often the beginners who get it first. Jane Alexander

Having honed Hollywoods highly insured bodies, The Ranch, known for its earthy fitness small, zesty-fresh plant-based portions and ferocious group hikes in nature has been airlifted to the sylvan surrounds of Palazzo Fiuggi. This frescoed haven outside Rome is best known for pristine medical attention and Heinz Becks carnivorous cuisine. The Ranch Italy at Palazzo Fiuggi is an unusual pairing but the proof is in the pudding. A 5.30am wake-up call heralds a stretch class followed by homemade granola and almond milk before a gruelling four-hour mountain hike that has some quitting after day one (theres more cardio and optional yoga later). Vegan fare such as macadamia Parmigiana is on the table throughout, with zero caffeine and alcohol. Daily deep tissue massages and restorative plunges into Kneipp pools take the edge off. For those who last the ride it is game-changing, with inches lost around midsections and bottoms hewn as lithe as lazio oak. Jemima Sissons

Healing Holidays can arrange a seven-night programme from 7,519 per person full board; healingholidays.com

Yoga as a gymnastic ego trip is losing its grip, while somatic movement, embodiment, fascial unwinding and pandiculation are edging in. At Yobaba Lounge in south-east France, embodiment pioneer Gertrud Keazor guides you to inhabit that soft animal of your body rather than bludgeoning you into the perfect Warrior II. Eyes are kept shut, all the better to enjoy birdsong and the soft breeze. Forget rigid adjustments follow your unfurling. You twist and twine around the mat, working deep into the fascia; its as much an emotional as a physical workout. Malabar Retreats centres on the Tibetan healing practice of Lu Jong at its outposts in Spain, Mozambique and Zimbabwe. Its a stringent practice but doesnt require bendiness or strength so works for beginners, or anyone sick of yoga-sculpt. Gone are the days of gurus, lineages and the same set of pre-defined poses, says trailblazer Gillie Sutherland, who runs retreats in Croatia and online workshops. Its about working with the bodys natural intelligence, wild and free. JA

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New stem cell therapy provides long-term brain protection against ALS – Study Finds

Posted: September 8, 2022 at 2:01 am

LOS ANGELES Cedars-Sinai researchers say a new stem cell therapy procedure allows them to protect patients with ALS, or Lou Gehrigs disease, and helps block muscle deterioration which normally occurs as a result of the fatal neurological disorder.

The Cedars-Sinai team successfully engineered and embedded protective proteins through the blood-brain barrier of patients with amyotrophic lateral sclerosis (ALS). The procedure increases hope that similar one-time treatments will greatly slow the diseases degenerative effects, including limb paralysis and the loss of ones ability to move, speak, or breathe. Researchers did not encounter any negative side-effects, while patients avoided leg paralysis following the transplant and replication of protein-producing stem cells from patients central nervous systems.

This breakthrough investigational therapy promotes the survival of motor neurons which typically degenerate in the spinal cord of patients with ALS. The researchers highlighted that none of the 18 people who underwent the stem cell gene therapy endured any serious side-effects after the transplantation.

Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that cant otherwise get through the blood-brain barrier, says corresponding author Clive Svendsen, PhD, professor of Biomedical Sciences and Medicine and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, in a media release.

The safety of the trial and a lack of side-effects among patients is receiving praise from numerous scientists in the ALS and neurological research community.

We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS, Svendsen says.

The researchers had several primary goals in this trial, which sought to show that stem cells engineered in Svendsens laboratory could produce the glial cell line-derived neurotrophic factor (GDNF), which help motor neurons pass signals between the brain and spinal cord. This process ultimately allows ALS patients to continue muscle movement which the disease typically destroys.

Prior to this recent study, neurological researchers feared there could be dire side effects or an inability to successfully bypass ALS patients blood-brain barrier using this therapeutic procedure. The blood-brain barrier, or BBB, serves as a structural and functional roadblock to potentially harmful microorganisms including parasites, viruses or bacteria in a persons bloodstream.

Because they are engineered to release GDNF, we get a double whammy approach where both the new cells and the protein could help dying motor neurons survive better in this disease, Svendsen added.

Researchers say they will soon build on the findings, published in the peer-reviewed journal Nature Medicine, including tests which target lower areas on the spinal cord and enrolling ALS patients in the study much earlier in their diagnosis.

We are very grateful to all the participants in the study, Svendsen concludes. ALS is a very tough disease to treat and this research gives us hope we are getting closer to finding ways to slow down this disease.

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$150 Million Gift Takes Stem Cell Research to New Heights – University of California San Diego

Posted: September 8, 2022 at 2:01 am

From left, T. Denny Sanford, Catriona Jamieson, MD, PhD, and Chancellor Pradeep K. Khosla celebrate the establishment of the UC San Diego Sanford Stem Cell Institute, made possible by a historic gift from Sanford.

Noted businessman and philanthropist T. Denny Sanford has committed $150 million in new funding to expand and, in some ways, quite literally launch stem cell research and regenerative medicine at University of California San Diego into new spaces and endeavors.

The gift will fund the new UC San Diego Sanford Stem Cell Institute and builds upon a $100 million gift in 2013 from Sanford that boldly established UC San Diego as a leader in developing and delivering the therapeutic promise of human stem cells special cells with the ability to develop into many different cell types and which, when modified and repurposed, have the potential to treat, remedy or cure a vast array of conditions and diseases.

Dennys previous generosity spurred discoveries in stem cell research and medicine at UC San Diego that are already benefiting countless patients around the world, said Chancellor Pradeep K. Khosla. His most recent gift adds to our portfolio of stem cell research conducted in Earths orbit that will help us better understand the progression of cancer cells and aging.

Sanfords gift to establish the Sanford Stem Cell Institute is the largest single gift to UC San Diego. This investment enables the team to dream beyond what is possible, said Sanford. The sky is no longer the limit.

In addition to his investment to create the Sanford Stem Cell Clinical Center at UC San Diego Health in 2013, Sanford established the T. Denny Sanford Institute for Empathy and Compassion in 2019, which focuses on research into the neurological basis of compassion, with application toward developing compassion and empathy-focused training for future generations of medical professionals. He also recently made a $5 million gift to support the Epstein Family Alzheimers Research Collaboration, a partnership between UC San Diego and the University of Southern California to spark new collaborative efforts to discover effective therapies for Alzheimers disease.

Sanford was also honorary co-chair of the Campaign for UC San Diego, which concluded in June 2022 having raised more than $3 billion exceeding its initial $2 billion goal. He was honored as a recipient of the 2014 Chancellors Medal, one of the universitys highest honors, in recognition of his exceptional service in support of the campus mission.

Stem cell research will be conducted in a laboratory bay located aboard the International Space Station, pictured here, in low-Earth orbit. Credit: NASA

The new UC San Diego Sanford Stem Cell Institute, under the direction of Catriona Jamieson, MD, PhD, Koman Family Presidential Endowed Chair in Cancer Research in the UC San Diego School of Medicine, will continue three existing stem cell programs at UC San Diego with three new programs.

The new programs to be established with Sanfords gift include:

Existing stem cell programs at UC San Diego in the Sanford Stem Cell Institute include:

We are thrilled to announce the establishment of the UC San Diego Sanford Stem Cell Institute with Denny Sanfords generous support, said Jamieson. This will allow us to keep pace with the growing need for regenerative and stem-cell based therapies and accelerate translational stem cell research and discoveries that will transform human health for years to come.

With three new programs established as part of the Sanford Stem Cell Institute, a key focus of the institute will be leveraging space as a new frontier for stem cell science. Exposure to radiation and microgravity in low-Earth orbit can simulate and speed up aging in stem cells, as well as their transformation into cancer cells. Space-related research may have applications that create better treatments for various cancers and diseases on earth, including blood cancers, as well as neurodegenerative diseases such as Alzheimers and Parkinsons.

To fuel sustained research and education in this promising area, Sanfords gift will establish the Sanford Stem Cell Institute STELLAR Endowed Chair in Regenerative Medicine, the Sanford Stem Cell Institute Endowed STELLAR Exploration Faculty Scholars and Fellows Fund, and the Sanford Stem Cell Institute STELLAR Exploration Discovery Fund.

UC San Diego already has expanded its research capacity in stem cell science to space efforts that will be further amplified with the recent gift.

In late 2021, UC San Diego worked with NASA, Space Tango and the JM Foundation to launch stem cells into space aboard a SpaceX Falcon 9 rocket to study stress-induced aging and how stem cells and their progeny transform into pre-cancer and cancer stem cells associated with leukemia and other blood cancers.

Allyson Muotri, PhD, with human organoid samples

In 2019, Alysson Muotri, PhD, professor of pediatrics and cellular and molecular medicine, and colleagues sent a payload of stem cell-derived human brain organoids to the International Space Station (ISS) orbiting almost 250 miles above Earth to study how these masses of cells organize into the beginnings of a functional brain in microgravity. The first-ever project of its type was dedicated to Sanford, a longtime supporter of Muotris work and others.

When I was designing these experiments, I realized how innovative and cutting edge they were, said Muotri. I thought Denny would be proud of this project, and that I should dedicate this first mission to him. Denny has been a cheerleader for the stem cell community. He is pushing all of us to speed discovery and translate it to help millions of people who suffer from different conditions that could be treated with stem cell-based therapies.

Since its inception in 2013, the Sanford Stem Cell Clinical Center at UC San Diego has yielded a three-fold return on investment by obtaining more than $312 million in funding, including $253.6 million in grants, $15.8 million in clinical trial contracts, $2.7 million in Advanced Cell Therapy Lab (ACTL) service charges and more than $40.2 million in philanthropy all with the goal of discovering new treatments to benefit patients.

Key successes include new pharmaceutical treatments Fedratinib, which was approved by the FDA for the treatment of myelofibrosis in 2019, and Glasdegib, FDA approved for acute myeloid leukemia in 2018.

Meanwhile, clinical trials are ongoing for Cirmtuzumab, a monoclonal antibody-based drug developed by Thomas Kipps, MD, PhD, Distinguished Professor of Medicine and deputy director of research at Moores Cancer Center at UC San Diego Health, and colleagues. Cirmtuzumab targets cancer stem cells and is being tested, alone and in combination with other drugs, to treat chronic lymphocytic leukemia and other blood cancers.

Stem cell research at UC San Diego has been a substantial beneficiary of the California Institute for Regenerative Medicine (CIRM), the states stem cell agency, created in 2004 with the approval of Proposition 71. UC San Diego researchers have garnered 116 awards totaling more than $227 million. Cirmtuzumab is named as a nod to CIRM and its support. In 2020, California voters passed Proposition 14 to continue CIRM operations and funding.

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Seattle biotech company is the first to receive approval to test B cell gene therapy in humans – GeekWire

Posted: September 8, 2022 at 2:01 am

Immusofts steps to delivering a treatment using engineered B cells. (Screen grab from Immusofts website)

Seattle biotech startup Immusoft has received approval to begin clinical trials of its novel strategy for treating genetic disease, the company announced Thursday. Immusoft says its the first to get permission to use engineered immune system cells called B cells in a human study.

The U.S. Food and Drug Administration (FDA) approved Immusofts Investigational New Drug Application for testing its immunotherapy for a rare, lethal childhood disease called MPS I.

An immune response includes a suite of players, and the B cells are responsible for producing the antibodies that stick to invading bacteria and viruses. The company is able to modify B cells into biofactories that instead crank out missing or non-functioning enzymes and proteins in the cells of patients.

This is a huge achievement for the company and a historic moment in the field of cell and gene therapies, said Sean Ainsworth, Immusofts CEO and chairman, in a statement.

The approach has potential advantages to current strategies for delivering treatments. Therapies that use a virus as its delivery mechanism can trigger immune responses that limit their effectiveness. Treatments using stem cells can have difficulties associated with chemotherapy and stem cell transplants.

Immusofts technology, called ISP-001, uses a patients own B cells, reprogramming them to make needed proteins. Other companies working on B cell therapies include Be Biopharma and Walking Fish Therapeutics.

I dont know if they are going to be successful, but its exciting for all of us that they have gotten permission to start a trial, researcher Richard James told MIT Technology Review. Jamess lab at the University of Washington is also working on B cell engineering.

The trial will be done at the University of Minnesota Medical School and led by Dr. Paul Orchard, a professor in the universitys Division of Pediatric Bone Marrow Transplantation.

Children with MPS I are not able to produce an essential enzyme that helps break down long-chain sugars inside cells. The sugars then build up in cells, causing progressive damage. Severe MPS1 occurs in about 1 in 100,000 births, and symptoms appear within a year.

Immusoft is interested in expanding its therapy to other rare diseases, as well as cardiovascular, autoimmune and central nervous system diseases.

In October 2021, the company announced a collaboration with pharma giant Takeda to develop treatments targeted to the nervous system in a deal worth potentially more than $900 million.

Immusoft was founded in 2009 and has raised more than $50 million in venture capital, according to PitchBook. In 2018, Ainsworth took over leadership from founder Matthew Scholz.

Scholz is now CEO of Oisn Biotechnologies, a startup developing preclinical therapies that target and kill damaged zombie cells. He is still on Immusofts board of directors.

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Seattle biotech company is the first to receive approval to test B cell gene therapy in humans - GeekWire

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Kite’s CAR T-cell Therapy Tecartus Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia…

Posted: September 8, 2022 at 2:01 am

SANTA MONICA, Calif.--(BUSINESS WIRE)--Kite, a Gilead Company (Nasdaq: GILD), today announced that the European Commission (EC) has approved its CAR T-cell therapy Tecartus (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL).

This approval makes Tecartus the first and only CAR T-cell therapy indicated for this population of patients, addressing a significant unmet medical need, said Christi Shaw, CEO, Kite. This is also the fourth indication in Europe for which a Kite cell therapy is approved, clearly demonstrating the benefits they offer to patients, especially those with limited treatment options.

ALL is an aggressive type of blood cancer; the most common form is B-cell precursor ALL. Globally, approximately 64,000 people are diagnosed with ALL each year. Half of adults living with ALL will relapse, and median overall survival (OS) with current standard-of-care treatments is approximately just eight months.

Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy and stem cell transplant, creating a significant burden on a patients quality of life, said Max S. Topp, MD, professor and head of Hematology, University Hospital of Wuerzburg, Germany. Patients in Europe now have a meaningful advancement in treatment. Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.

The approval is supported by data from the ZUMA-3 international multicenter, single-arm, open-label, registrational Phase 1/2 study of adult patients (18 years old) with relapsed or refractory ALL. This study demonstrated that 71% of the evaluable patients (n=55) achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) with a median follow-up of 26.8 months. In an extended data set of all pivotal dosed patients (n=78) the median overall survival for all patients was more than two years (25.4 months) and almost four years (47 months) for responders (patients who achieved CR or CRi). Among efficacy-evaluable patients, median duration of remission (DOR) was 18.6 months.

Among the patients treated with Tecartus at the target dose (n=100) safety results were consistent with the known safety profile for Tecartus. Grade 3 or higher cytokine release syndrome (CRS) and neurologic adverse reactions occurred in 25% and 32% of patients, respectively, and were generally well managed.

About ZUMA-3

ZUMA-3 is an ongoing international multicenter (US, Canada, Europe), single arm, open label, registrational Phase 1/2 study of Tecartus in adult patients (18 years old) with ALL whose disease is refractory to or has relapsed following standard systemic therapy or hematopoietic stem cell transplantation. The primary endpoint is the rate of overall complete remission or complete remission with incomplete hematological recovery by central assessment. Duration of remission and relapse-free survival, overall survival, minimal residual disease (MRD) negativity rate, and allo-SCT rate were assessed as secondary endpoints.

About Acute Lymphoblastic Leukemia

ALL is an aggressive type of blood cancer that develops when abnormal white blood cells accumulate in the bone marrow until there isnt any room left for blood cells to form. In some cases, these abnormal cells invade healthy organs and can also involve the lymph nodes, spleen, liver, central nervous system and other organs.

About Tecartus

Please see full FDA Prescribing Information, including BOXED WARNING and Medication Guide.

Tecartus is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITIES

Cytokine Release Syndrome (CRS), including life-threatening reactions, occurred following treatment with Tecartus. In ZUMA-2, CRS occurred in 92% (72/78) of patients with ALL, including Grade 3 (Lee grading system 1) CRS in 26% of patients. Three patients with ALL had ongoing CRS events at the time of death. The median time to onset of CRS was five days (range: 1 to 12 days) and the median duration of CRS was eight days (range: 2 to 63 days) for patients with ALL.. Among patients with CRS, the key manifestations (>10%) were similar in MCL and ALL and included fever (93%), hypotension (62%), tachycardia (59%), chills (32%), hypoxia (31%), headache (21%), fatigue (20%), and nausea (13%). Serious events associated with CRS included hypotension, fever, hypoxia, tachycardia, and dyspnea.

Ensure that a minimum of two doses of tocilizumab are available for each patient prior to infusion of Tecartus. Following infusion, monitor patients for signs and symptoms of CRS daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility, and for four weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

Neurologic Events, including those that were fatal or life-threatening, occurred following treatment with Tecartus. Neurologic events occurred in 81% (66/82) of patients with MCL, including Grade 3 in 37% of patients. The median time to onset for neurologic events was six days (range: 1 to 32 days) with a median duration of 21 days (range: 2 to 454 days) in patients with MCL. Neurologic events occurred in 87% (68/78) of patients with ALL, including Grade 3 in 35% of patients. The median time to onset for neurologic events was seven days (range: 1 to 51 days) with a median duration of 15 days (range: 1 to 397 days) in patients with ALL. For patients with MCL, 54 (66%) patients experienced CRS before the onset of neurological events. Five (6%) patients did not experience CRS with neurologic events and eight patients (10%) developed neurological events after the resolution of CRS. Neurologic events resolved for 119 out of 134 (89%) patients treated with Tecartus. Nine patients (three patients with MCL and six patients with ALL) had ongoing neurologic events at the time of death. For patients with ALL, neurologic events occurred before, during, and after CRS in 4 (5%), 57 (73%), and 8 (10%) of patients; respectively. Three patients (4%) had neurologic events without CRS. The onset of neurologic events can be concurrent with CRS, following resolution of CRS or in the absence of CRS.

The most common neurologic events (>10%) were similar in MCL and ALL and included encephalopathy (57%), headache (37%), tremor (34%), confusional state (26%), aphasia (23%), delirium (17%), dizziness (15%), anxiety (14%), and agitation (12%). Serious events including encephalopathy, aphasia, confusional state, and seizures occurred after treatment with Tecartus.

Monitor patients daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility and for four weeks following infusion for signs and symptoms of neurologic toxicities and treat promptly.

REMS Program: Because of the risk of CRS and neurologic toxicities, Tecartus is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta and Tecartus REMS Program which requires that:

Hypersensitivity Reactions: Serious hypersensitivity reactions, including anaphylaxis, may occur due to dimethyl sulfoxide (DMSO) or residual gentamicin in Tecartus.

Severe Infections: Severe or life-threatening infections occurred in patients after Tecartus infusion. Infections (all grades) occurred in 56% (46/82) of patients with MCL and 44% (34/78) of patients with ALL. Grade 3 or higher infections, including bacterial, viral, and fungal infections, occurred in 30% of patients with ALL and MCL. Tecartus should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after Tecartus infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 6% of patients with MCL and 35% of patients with ALL after Tecartus infusion and may be concurrent with CRS. The febrile neutropenia in 27 (35%) of patients with ALL includes events of febrile neutropenia (11 (14%)) plus the concurrent events of fever and neutropenia (16 (21%)). In the event of febrile neutropenia, evaluate for infection and manage with broad spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, life-threatening and fatal opportunistic infections have been reported. The possibility of rare infectious etiologies (e.g., fungal and viral infections such as HHV-6 and progressive multifocal leukoencephalopathy) should be considered in patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

Prolonged Cytopenias: Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and Tecartus infusion. In patients with MCL, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 55% (45/82) of patients and included thrombocytopenia (38%), neutropenia (37%), and anemia (17%). In patients with ALL who were responders to Tecartus treatment, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 20% (7/35) of the patients and included neutropenia (12%) and thrombocytopenia (12%); Grade 3 or higher cytopenias not resolved by Day 60 following Tecartus infusion occurred in 11% (4/35) of the patients and included neutropenia (9%) and thrombocytopenia (6%). Monitor blood counts after Tecartus infusion.

Hypogammaglobulinemia: B cell aplasia and hypogammaglobulinemia can occur in patients receiving treatment with Tecartus. Hypogammaglobulinemia was reported in 16% (13/82) of patients with MCL and 9% (7/78) of patients with ALL. Monitor immunoglobulin levels after treatment with Tecartus and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement.

The safety of immunization with live viral vaccines during or following Tecartus treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least six weeks prior to the start of lymphodepleting chemotherapy, during Tecartus treatment, and until immune recovery following treatment with Tecartus.

Secondary Malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

Effects on Ability to Drive and Use Machines: Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following Tecartus infusion. Advise patients to refrain from driving and engaging in hazardous activities, such as operating heavy or potentially dangerous machinery, during this period.

Adverse Reactions: The most common non-laboratory adverse reactions ( 20%) were fever, cytokine release syndrome, hypotension, encephalopathy, tachycardia, nausea, chills, headache, fatigue, febrile neutropenia, diarrhea, musculoskeletal pain, hypoxia, rash, edema, tremor, infection with pathogen unspecified, constipation, decreased appetite, and vomiting. The most common serious adverse reactions ( 2%) were cytokine release syndrome, febrile neutropenia, hypotension, encephalopathy, fever, infection with pathogen unspecified, hypoxia, tachycardia, bacterial infections, respiratory failure, seizure, diarrhea, dyspnea, fungal infections, viral infections, coagulopathy, delirium, fatigue, hemophagocytic lymphohistiocytosis, musculoskeletal pain, edema, and paraparesis.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Gilead and Kite to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving Tecartus; the risk that physicians may not see the benefits of prescribing Tecartus for the treatment of blood cancers; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Tecartus including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com.

Kite, the Kite logo, Tecartus and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.

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Kite's CAR T-cell Therapy Tecartus Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia...

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Sarepta Therapeutics Announces Recipients of Route 79, The Duchenne Scholarship Program, for … – The Bakersfield Californian

Posted: September 8, 2022 at 2:00 am

- Recipients include 15 individuals living with Duchenne and five siblings in Duchenne families

CAMBRIDGE, Mass., Sept. 07, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced twenty recipients of Route 79, The Duchenne Scholarship Program for the 2022-2023 academic year. The Program was created in 2018 to recognize exceptional individuals living with Duchenne muscular dystrophy as they pursue their post-secondary education. Now in its fifth year, the Program was expanded to include siblings of individuals with Duchenne in recognition of the impact that a diagnosis of Duchenne may have on the entire family. Recipients of the scholarship are chosen by an independent selection committee composed of Duchenne community members, who consider each applicants community involvement and personal essay. Each student will receive a scholarship of up to $5,000.

On behalf of Sarepta and the selection committee, we are thrilled to announce the recipients of Route 79, The Duchenne Scholarship Program, for the 2022-2023 academic year. These twenty outstanding students are exemplary in their commitment and dedication to their studies. In addition to conveying their intellectual curiosity, the essays from this years recipients spoke to the power and strength of community and the importance of advocating for growth and change at a personal and societal level, said Diane Berry, senior vice president, Global Health Policy, Government and Patient Affairs, Sarepta. We are honored to support these young adults as they pursue their educational goals, and we wish them great success in the school year ahead and wherever their academic journey takes them.

2022 Recipients Individuals Living with Duchenne

Porter Aydelotte, California State University, Long Beach

Jared Conant, University of Southern Maine

Aiden Fecteau, Eastern Connecticut State University

Bryson Foster, University of North Carolina, Charlotte

Yuvaraj Gambhir, University of Pennsylvania

Maanav Gupta, University of Houston

Ethan Higginbotham, Wichita State University

Elliott Johnson, Lebanon Valley College

Joshua Jurack, James Madison University

John McConnell, Boise State University

Josh Pflueger, Texas Christian University

Robert Sullivan, John Carroll University

Tayjus Surampudi, Harvard University

Joseph Ware, Liberty University

Jack Wolf, University of Akron, Main Campus

2022 Recipients Siblings in Duchenne Families

Luke Kieser, Indiana Institute of Technology

Grace Lee, University of San Diego

Zoie Liska, Wichita State University

Dylan Malone, University of Mississippi Medical Center

Reese Manderfield, University of Iowa

In addition to application review by the independent committee, submissions are de-identified for the voting panel with no indication of whether the candidate has received, or plans to receive, a Sarepta therapy.

AboutRoute 79, TheDuchenne Scholarship Program

Route 79, TheDuchenne Scholarship Program is designed to help students with Duchenne and siblings of individuals living with Duchenne pursue their post-secondary educational goals. There are 79 exons in the dystrophin gene impacted by Duchenne, and the route traveled by every person impacted by Duchenne is distinct. Sareptas goal through this program is to acknowledge and support individuals with Duchenne and their siblings, who are mapping out their future via educational pursuits.Scholarship recipients are chosen by an independent committee of Duchenne community members based on an applicants community involvement, personal essay, and recommendation letter. Additional information is available at https://www.sarepta.com/route79.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit http://www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Investor Contact:

Ian Estepan, 617-274-4052

iestepan@sarepta.com

Media Contact:

Tracy Sorrentino, 617-301-8566

tsorrentino@sarepta.com

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Sarepta Therapeutics Announces Recipients of Route 79, The Duchenne Scholarship Program, for ... - The Bakersfield Californian

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‘Record’ Gains in Bringing Research Closer to the Patient – Applied Clinical Trials Online

Posted: September 8, 2022 at 2:00 am

Experts discuss solving the EHR-compatibility puzzle in point-of-care trials.

Editors note: Industry recognition for the clinical research as a care option, or CRAACO, movement goes to The Conference Forum, which has hosted the conference of the same name, for seven years. This years CRAACO event was held Sept. 12 and Applied Clinical Trials, at the time of this writing, is looking forward to attending and learning more from many of the speakers featured in this article.

At the Icahn School of Medicine at Mount Sinai, researchers are laying the groundwork for future massive genotype-based studies. Within three to six years, the database is expected to hold the sequenced DNA samples of one million peopleall patients of the Mount Sinai Hospital System.

In Oregon and California, researchers wanted to see if people who were overdue for a colon cancer screening test would take one if they were mailed a FIT test. It took one year to identify 41,193 people in 26 federally qualified health clinics.1

And in the Northwell Health System, researchers culled their electronic health record (EHR) system for patients with specific COVID-19 symptoms to help them determine whether the use of famotidine would shorten the number of days a COVID patient contended with symptoms. They found 55 fitting the bill within six weeks.2

At the University of California, San Francisco, and across the country at Temple University Hospital, oncologist researcher Laura Esserman, MD, MBA, and pulmonologist Gerard Criner, MD, respectively, have been using their EHR systems for years to run pragmatic trials and to find eligible clinical trial patients in their respective clinics.3,4 Once found, the medical teams embed the trial into patient care.

If you are wondering if this article concerns EHRs and compatibility, youd be correct. If you are thinking it concerns ways in which clinical trials can be run with less money and fewer data points, youd also be accurate. And if you think its about improving patient care where they receive careall while advancing researchyou are spot on.

What these researchers are doing bears little resemblance to the classic, rigidly run, interventional clinical trial. Trial phases blend into one another. In some cases, molecules under inspection that arent working are eliminated as early as possible from the trial; molecules that do work are tested under more scrutiny. With clinical trials, time means money.

A key point: All enrolled patients were found after serious mining into the providers EHR. So yes, point of care works in large healthcare establishments, with fluid EHR systems. So yes again, point of care is not a routine approach in most trials. A serious problem, says Amy Cramer, director of global product development strategic partnerships at Pfizer and the steering committee board co-chair for the HL7 FHIR Accelerator Vulcan, is that most trials are still conducted using paper processes. Researchers need the data before the trial is started, she adds.

It is all about the technology, Christina Brennan, MD, vice president, clinical research at Northwell Health, tells Applied Clinical Trials. We wouldnt have been able to do [the famotidine trial] without the EHR system, she says. Everything Northwell could determine about its potential recruitment prospects came from its system, as did all inputted data that led to results. We screened thousands of patients for eligibility.

At Temple University Hospital, running trials and treating patients have gone hand in hand for a few years. Our clinical research program is not separate from our clinical care or our academic mission, said Criner, also chair and professor, Thoracic Medicine and Surgery, Lewis Katz School of Medicine at Temple University; and director, Temple Lung Center, in a published interview3, who also reviewed this included information for accuracy. If you embed it into your care program and your care program has to address what the needs of the patient community you treat are, you cant help but make everything betteryour research, clinical care, and training.

As to what this clinical trial approach is called, there is no universal name. Brennan says she didnt think point of care was applicable, and preferred clinical research as a care option, or precision/personalized medicine.

According to FDAwhich, by the way, is a big supporter of embedding clinical research at the point of carePoint of care trials take place in a clinical practice environment and make use of clinical infrastructure and personnel for trial-related activities. A successful trial, an agency spokesperson continued, may shorten the time needed to obtain safety and efficacy data for medical products.

But do not think these trials are allowed to confuse real-world data (RWD) with real-world evidence (RWE). FDA, in its 2018 framework document,5 defined RWD as the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources, and RWE is the clinical evidence regarding a medical products use and potential benefits or risks derived from analysis of RWD.

Says researcher Rachele Hendricks-Sturrup, DHSc, research director for real-world evidence at the Margolis Health Center at Duke University: Healthcare data is not real-world evidence until it is combined with real-world data.

Taking the 30,000-foot view, Jodyn Platt, PhD, MPH, an assistant professor of learning health sciences at the University of Michigan, says health care should be viewed as a point of learning. Theres a fine line between quality improvement and research in order to learn from that data to give better care.

But it could be worth it, Platt says. If researchers have the opportunity to use a system that allows them to see what is done correctly or not, we have the potential for making the [healthcare system] better for everyone, she says. If we can think strategically, we can make the process better.

Esserman, who built her approach to breast cancer trials in the early 2010s, described how the process works at the UCSF Breast Cancer Center. All registration data are collected and cleaned within 24 hours.4

Each patients information regarding response to the therapy is used to build on to the next patient. This is all about finding early indicators of response and eliminating data detritus, which, says Esserman, clouds results.

The idea, she adds, is to have real-time data collection. All data sets must be complete before randomization, Platt says. We are trying to focus on the data that inform the primary endpoints, so that we are less burdened with data of ancillary interest.

She elaborates that most data, 90%, collected in trials is irrelevant because it is never used or not germane to the primary endpoint. And since all data collection requires time and money, more thoughtful decisions about what to collect matters.

It is a model other are adopting or participating in, including FDA and the National Cancer Institute.7

In Northwells double-blind, placebo-controlled study on famotidine, researchers collected 1,358 data points from 55 patients with COVID; one arm got the famotidine, the other, placebo. Those in the control arm improved three days more quickly. All samples and all treatment were administered in the patients homes. The cost of the trial was less than $1.5 million.2 The success of the study and its cheaper cost have convinced Northwell that the model is worth incorporating into their trial formats. Along with this more patient-centric approach, Brennan says that hybrid trials are being requested more by sponsors.

Between 2014 through 2016, Mount Sinai used EHRs to find 2,050 patients of West-African ancestry with hypertension, no chronic kidney disease, and genotyped them for the APOL1 risk variant, which could be deadly for those subgroups.6 Once patients were found through the EHRs, study coordinators mailed invitations and then followed up with phone calls of those who declined, and intercepted potentially eligible patients at clinics when they were hard to reach, according to the study. The point of the trial was to quickly return genetic results to a randomized group to see how patient outcomes would be affected.

The intervention group reduced their blood pressure and adopted more healthy behaviors. The high-risk patients dropped their systolic blood pressure by 3.6%; the controls dropped by 1.3%.

These results may support an approach of broad implementation of genetic medicine in primary care for certain use cases, concluded Girish N. Nadkarni, MD, MPH, Irene and Dr. Arthur M. Fishberg professor of Medicine, Icahn School of Medicine at Mount Sinai, et al., in the study.6

As to the question of just how common point of care trial research actually is, everyone interviewed had the same answerit isnt.

There is a lot of activity in this space. But it is fragmented, as is our healthcare system, says Platt. Her specialty is studying trust in the US healthcare system. Developing measures of trust to monitor how trial enrollees are faring is in the works, she she adds. It is a huge issue because of digital care in the home. What happens to the data if third parties are involved, or if a startup company that was monitoring patient progress dissolves? How does your data live with this? asks Platt.

She and her staff are starting research in artificial intelligence and how patients trust the technology in their healthcare systems. That is a key research questionits a new form of technology that has little oversight, from institutions and government. We need to understand what the public feels like when they have a stake in the field.

In May, the Duke University Margolis Center for Health Policy published a white paper on point of care-incorporated trials.8 It defined one aspect of the point-of-care scenario as: Using an EHR for enrollment, data collection, and randomization; running the trial where patient care occurs; and combining research and clinical care delivery workflows. The focus is on the trial participants, says Hendricks-Sturrup, a co-author. We want to achieve better screening, consent, randomization, and data collection, then incorporating those finds into routine care.

The difference between other trial designs and those that use point of care, says Hendricks-Sturrup, is that point of care is designed to know if the therapy is efficacious across all populations.

Margolis is working to resolve issues that could impede point-of-care progress, such as who runs the trial, who should pay for it, and how can data be collected and handled in an easy fashion across different systems? A major point is not adding burden to providers. We are working to tackle policy, says Trevan Locke, PhD, assistant research director for Margolis.

As for the blip, researchers for the colon cancer screening trial learned that buy-in from staff involved in the trial is essential. Staff at the various federally qualified health clinics didnt all do as the researchers asked, like installing a piece of software into their EHRs to find and track possible participants. Researchers stated: For most of the participating health centers, [this trial] represented the first time EHR tools were used to deliver cancer screening services outside the clinic. Implementation might have increasedwith experience.1

Alexander Charney, MD, PhD, associate professor, departments of psychiatry and genetics and genomic sciences, and co-director of the Mount Sinai Million Health Discoveries Program, says that as it acquires patient samples, it is gaining approvals for future trials. No studies are established yet, he says.

But lets fast-forward five years, Charney continues. A sponsor wants to run a trial comparing therapy X to therapy Y, when half of the patients have a certain genetic variant. If a researcher wants to do that trial, five years from now, patients will already have consented, he adds. We have laid the groundwork.

All 17 hospitals and clinics in the Mount Sinai Health System will be recruiting for this project, which is being underwritten by Regeneron. The volume of patients and unification of Mount Sinais operations make the enrollment goal possible, says Charney. His fellow co-director, Nadkarni, says enrollment is going slowly for a purpose.

We want to make sure systems are good to go, he tells Pharm Exec, and that safeguards are working, like ensuring patients are not duplicated in the system. The platform, he notes, meets all NIH and Department of Defense security standards.

Partnering with Regeneron, says Charney, was key to getting the project off the ground. There is no other way to sequence the genome of one million people, he adds. Regeneron, notes Nadkarni, will sequence the DNA samples, and then return the patient data.

At Northwell, Brennan said that as each trial request comes in, the team is looking at whether an embedded study can work. Of the 2,000 trials in motion, 5% to 8% are designed as a hybrid approach. Despite all the talk about embedded, i.e., point of care, few are being run that way.

But, she adds, We dont want to go back to the former ways and traditional methods of running a trial. When we saw how to pivot in COVID, we were able to [be flexible].

Christine Bahls is a freelance writer for medical, clinical trials, and pharma information.

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'Record' Gains in Bringing Research Closer to the Patient - Applied Clinical Trials Online

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Metagenomi Announces Participation in September Investor and Industry Conferences – Business Wire

Posted: September 8, 2022 at 2:00 am

EMERYVILLE, Calif.--(BUSINESS WIRE)--Metagenomi, a gene editing company with a versatile portfolio of next-generation CRISPR gene editing tools, today announced its participation in the following investor and industry conferences:

Citis 17th Annual BioPharma ConferenceCompany Panel titled Private Co-Panel: Genetic Medicine on September 7, 4:20 - 5:05 p.m. EDT11 meetings with institutional investorsParticipants: Brian C. Thomas, Ph.D., CEO and founder, and Simon Harnest, CIO, SVP Strategy

Wells Fargo Healthcare ConferenceCorporate presentation with Q&A on September 9, 10:25 - 10:55 a.m. EDT11 meetings with institutional investorsParticipants: Brian C. Thomas, Ph.D., CEO and founder, and Simon Harnest, CIO, SVP Strategy

Bairds 2022 Global Healthcare ConferenceCorporate presentation with Q&A on September 13, 9:40 - 10:10 a.m. EDTParticipant: Simon Harnest, CIO, SVP Strategy

GENedges GEN The State of Biotech Virtual SummitPre-recorded presentation, September 21-22Participant: Brian C. Thomas, Ph.D., CEO and founder

Jefferies Cell and Genetic Medicine SummitCorporate presentation with Q&A on September 3011 meetings with institutional investorsParticipant: Simon Harnest, CIO, SVP Strategy

About Metagenomi

Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.

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Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy – Yahoo Finance

Posted: September 8, 2022 at 2:00 am

Sarepta Therapeutics, Inc.

CAMBRIDGE, Mass., Sept. 06, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on SRP-5051 (vesleteplirsen), the Companys investigational, next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. After discussions with FDA and as part of the lift, Sarepta will adjust the global trial protocol to include expandedmonitoringof urine biomarkers.

The hold in Part B of Study 5051-201, also known as MOMENTUM, followed a serious adverse event of hypomagnesemia. Information was provided by the Company to FDA to assess the adequacy of the risk mitigation and safety monitoring plan.

We would like to thank FDA for working closely with us to expeditiously resolve this clinical hold. We will implement the changes in the protocol to resume dosing in the U.S. as quickly as possible, said Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta Therapeutics. Our monitoring plan is designed to mitigate the risks of hypomagnesemia. MOMENTUM has continued enrolling participants outside the U.S., and we remain on track to complete enrollment by the end of 2022. Sarepta is committed to the SRP-5051 program and excited about the PPMO platform as a next-generation exon-skipping approach for the treatment of Duchenne.

About SRP-5051 (vesleteplirsen) SRP-5051 is an investigational agent using Sareptas PPMO chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally shortened, functional dystrophin protein. PPMO is Sareptas next-generation chemistry platform designed around a proprietary cell-penetrating peptide conjugated to the PMO backbone, with the goal of increasing tissue penetration, increasing exon skipping, and significantly increasing dystrophin production. Around 13% of DMD patients have mutations that make them amenable to skipping exon 51. If successful, the PPMO offers the potential for improved efficacy and less frequent dosing for patients.

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About MOMENTUM (Study SRP-5051-201) MOMENTUM is a Phase 2, multi-arm, ascending dose trial of SRP-5051, infused monthly and will assess dystrophin protein levels in skeletal muscle tissue following SRP-5051 treatment. The trial will enroll up to 60 participants, both ambulant and non-ambulant, between the ages of 7 to 21 at sites in the U.S., Canada, and the European Union. The trial will also assess safety and tolerability.

In 2021, the Company announced results from Part A of MOMENTUM showing that after 12 weeks, 30 mg/kg of SRP-5051 dosed monthly resulted in 18 times the exon skipping and eight times the dystrophin production as eteplirsen, dosed weekly for 24 weeks. Reversible hypomagnesemia was identified in patients taking SRP-5051. The protocol for Part B of MOMENTUM includes magnesium supplementation and monitoring of magnesium levels.

More information can be found on http://www.clinicaltrials.gov.

About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding adjustments to our global trial protocol for SRP-5051; the potential benefits of PPMO and SRP-5051; our approach to monitoring and managing hypomagnesemia; and our expected timelines, plans, and milestones, including completing enrollment of Part B of MOMENTUM by the end of 2022, and resuming screening and dosing in the U.S. as quickly as possible.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical studies and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the ongoing COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2021, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Source: Sarepta Therapeutics, Inc.

Investor Contact: Ian Estepan, 617-274-4052iestepan@sarepta.com

Media Contact: Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy - Yahoo Finance

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