Double Duty: Early Research Reveals how a Single Drug Delivers Twice the Impact in Fragile X – URMC

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Like many neurological diseases, theres a lot we dont understand about fragile X syndrome. But, after studying the disorder for several years, Lynne Maquats lab knew two important things: the enzyme AKT, which plays a key role in cell growth and survival, and the quality control pathway known as NMD (nonsense-mediated mRNA decay), are both in overdrive in fragile X.

In a new study in the journal Molecular Cell, the team reveals how these two major players interact, highlighting a complex molecular dance that could inform the development of future treatments for fragile X syndrome.

Two paths to pursue

AKT is a hub for cell signaling, helping cells communicate about important processes like cell growth, proliferation and protein production. When cells are stressed for example, in cancer, diabetes, heart disease and neurological disorders, including fragile X AKT can send too many (or too few) signals or messages as part of a cell survival mechanism.

NMD is like a molecular guide that helps our cells make smart decisions that (in most cases) improve cellular function and contribute to good health. For example, NMD supports gene expression by flagging and destroying mRNAs (messenger RNAs) that are carrying faulty genetic instructions that could lead to disease. It also helps our cells adjust to changes in development and in their environment, andmore rapidly respond to certain stimuli.

Co-lead study authors Hana Cho, Ph.D., and Elizabeth Abshire, Ph.D., discovered how AKT and NMD interact in the context of fragile X:

Drug double whammy

Taking these findings a step further, the team treated the neural stem cells that mimic fragile X syndrome with a drug called Afuresertib, which inhibits AKT and is currently being tested in phase 1 and 2 clinical trials for several types of cancer. They found that blocking AKT in the fragile X cells not only decreased its activity, but decreased NMD, as well. The cells acted more like typical, non-disease cells when AKT was inhibited.

Normalizing two major pathways that contribute to fragile X syndrome is an exciting development, and using a drug that has already been through early clinical trials and that has been shown to be safe in patients puts us a step ahead, as opposed to starting from scratch with a brand new molecule, says Abshire, a postdoctoral fellow in the Maquat lab. There is still a lot we dont know about how AKT and NMD interact, because they are both massive pathways that influence and regulate multiple activities in cells, but this work provides good direction.

Next steps in the research include taking drugs like Afuresertib and testing them in a mouse model of fragile X to determine if what the team found in cells (AKT goes down and NMD goes down) also occurs in a living organism.

Drilling down on disease mechanism

AKT is stimulated or spurred into action by insulin. This study is the first to show that extracellular signaling (something that happens outside the cell, like an increase in insulin) changes the identity of a mark called the exon junction complex or EJC. Discovered by Lynne E. Maquat, Ph.D., founding director of the Center for RNA Biology at the University of Rochester, the EJC promotes NMD when certain conditions are met. Cho and Abshire showed that AKT is unexpectedly a member of the complex of proteins that constitute the EJC, which is important for normal gene expression.

By revealing a new mechanism by which AKT-signaling alters NMD and gene expression, we have a more complete understanding of disease mechanism. The more we know about this important signaling pathway, the more we can think about targets to suppress its hyperactivity, said Maquat, corresponding study author and the J. Lowell Orbison Endowed Chair and Professor ofBiochemistry and Biophysicsat the University of Rochester School of Medicine and Dentistry.This adds another aspect to how we can understand dysregulated pathways in diseases like fragile X and cancer when we are thinking about drugs.

In the study, the team also details a new tool that they developed for screening potential drugs that inhibit NMD, which is hyperactivated in fragile X and a number of cancers.

In addition to Maquat, Cho and Abshire, Maximilian W. Popp and Christoph Prschel, also of the University of Rochester School of Medicine and Dentistry, and Joshua L. Schwartz and Gene W. Yeo, of the University of California-San Diego, contributed to the research. The research was funded by an R01 to Maquat and an R21 to Prschel, both from the National Institutes of Health, and by a University of Rochester Provosts award to Maquat and Prschel.

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Double Duty: Early Research Reveals how a Single Drug Delivers Twice the Impact in Fragile X - URMC

New Technology Helps Reveal Inner Workings of Human Genome – Weill Cornell Medicine Newsroom

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Weill Cornell Medicine and New York Genome Center researchers, in collaboration with Oxford Nanopore Technologies, have developed a new method to assess on a large scale the three-dimensional structure of the human genome, or how the genome folds. The genome is the complete set of genetic instructions, DNA or RNA, enabling an organism to function.

Using this method, the researchers demonstrated that cell function, including gene expression, may be affected by groups of simultaneously interacting regulatory elements in the genome rather than pairs of these components. Their findings, published May 30 in Nature Biotechnology, may help shed light on the relationship between genome structure and cellular identity.

Knowing the three-dimensional genome structure will help researchers better understand how the genome functions, and particularly how it encodes different cell identities, said senior author Dr. Marcin Imieliski, associate professor of pathology and laboratory medicine and computational genomics in computational biomedicine at Weill Cornell Medicine and a core member of the New York Genome Center. The ways that weve had to study genome structure have given us amazing insights, but there have also been key limitations, he said.

For example, previous technology to assess the genomes three-dimensional structure has allowed researchers to study how frequently two loci, or physical locations on the genome, interact with one another. Traditionally, pairs of loci called enhancers and promoterscomponents in the genome that interact with one another to influence gene expressionhave been observed.

Information about these pairings offers incomplete insight into genome structure and function. For instance, linking a folding pattern to how the genome encodes for a specific cell identitylike a liver, lung or epithelial cellhas been difficult, said Dr. Imieliski, who is also a member of the Englander Institute for Precision Medicine and the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine. Scientists have theorized that this folding influences gene expression. But how cell types are encoded, particularly in the structure of DNA, has been a mystery, he said.

Dr. Imieliski and his research team, including first author Aditya Deshpande, a recent graduate of the Tri-Institutional Ph.D. Program in Computational Biology & Medicine working in Dr. Imieliskis lab, developed a new genome-wide assay and algorithm that allows them to study groups of loci, not just pairs.

They adapted a traditional technology, Hi-C (chromatin conformation capture), which assesses a mixture of DNA and protein to analyze three-dimensional genome structure, to nanopore sequencing, or the high-throughput sequencing of long, continuous strands of DNA molecules. The resulting assay, which the researchers called Pore-C, enabled them to observe tens of millions of three-dimensional locus groupings.

They also developed statistical methods to determine which locus groupings were important, based on whether they interacted cooperatively to affect gene expression. Many three-dimensional interactions of the genome are not important, Dr. Imieliski said. Our analytic methods help us prioritize the group interactions that are likely to matter for genome function. As a key finding of the study, the researchers found that the most significant cooperative groupings of DNA elements occurred around genes associated with cell identity.

Future experiments will explore which specific groupings of genomic components are essential for various aspects of cell identity. The new technology may also help researchers to understand how stem cells, the immature, master cells of the body, differentiate into different cell types.

In addition, researchers may be better able understand abnormalities in cancer cells. In the future, this technology may be really helpful in understanding how cancer cell genomes are rearranged, and how those rearrangements drive the altered cell identities that enable cancers to grow and spread Dr. Imieliski said.

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New Technology Helps Reveal Inner Workings of Human Genome - Weill Cornell Medicine Newsroom

HIV drug may improve cognitive function in people with Down syndrome – Study Finds

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BARCELONA, Spain A drug for treating HIV may also have the ability to help individuals with Down syndrome who deal with cognitive impairment. Researchers say tests on mice reveal that lamivudine improves cognition, which is a key problem for people with the genetic condition.

Lamivudine is a prescription medication approved for use in the United States to treat HIV infections in both adults and children. Researchers are hoping to start clinical trials in human patients in a near future.

People with the condition have an extra chromosome in their bodies. Typically, a child only has 46 chromosomes at birth. However, babies with Down syndrome have an extra copy of the 21st chromosome.

Unfortunately, this copy leads to children having mild to moderate intellectual disability, including problems with memory, speech, and attention span. During adulthood, these individuals also experience accelerated aging, which leads to cognitive decline more rapidly than in other older adults.

This puts people with Down syndrome at a much higher risk for the development of Alzheimers disease later in life. Specifically, researchers say chromosome 21 carries a gene amyloid precursor protein (APP) that creates amyloid proteins. Previous studies have shown that these proteins build up in the brain and lead to declining mental performance.

Amyloid accumulation is a common problem in most adult Down syndrome patients over the age of 40.

Currently, one of the few options available to help people with Down live independently is a psychosocial intervention like cognitive stimulation therapy. Study authors note that there are no drug treatments that can do this at the moment.

However, scientists are now looking at targeting retrotransposons in Down syndrome research. Retrotransposons are segments of DNA which change location inside a genome. The segments do this by making RNA copies of themselves, which then jump into another location on a DNA strand. Through this process, retrotransposons are able to insert themselves into specific genomes and position themselves in gene-promoting regions which have a connection to neurodegenerative diseases. This actually enhances their activity in the body.

The speed at which retrotransposition takes place also increases as someone gets older and more cells die off. This is where the connection to HIV comes in. Retrotransposons are similar to the virus, as they replicate rapidly inside cells.

Since lamivudine helps to inhibit HIV from replicating, the researchers examined the drugs ability to do the same with retrotransposons in mice.

Both HIV and retrotransposons need the same molecule to make copies of themselves: the reverse transcriptase enzyme, explains Dr. Bonaventura Clotet, Director of the IrsiCaixa AIDS Research Institute, in a media release.

We know that lamivudine, a reverse transcriptase inhibitor used against HIV, was shown in aged mice to decrease the activation of retrotransposons which could be linked to age-associated disorders. Therefore, we thought that it could be useful to counteract the cognitive impairment associated with Down syndrome.

The team used Ts65Dn mice, which are the most studied Down syndrome animal models available for research. Over four months, one group of mice received lamivudine treatments, while a control group only consumed a placebo (water).

During behavioral experiments testing their motor activity, memory, and levels of anxiety, the team found mice on lamivudine enjoyed greater levels of cognitive function. Researchers believe the drug is targeting at least one variant of the APP gene.

Our work aims to support people with Down syndrome and their families by providing them more options to live independent lives, particularly those affected by early-stage Alzheimers disease, says Dr. Mara Dierssen, researcher at the Centre for Genomic Regulation in Spain.

We still need pharmacological treatments that consistently help improve memory, attention and language functions, or prevent cognitive decline associated with ageing. This study is one step aiming to change that, revealing retrotransposition as an interesting mechanism to pursue not only in ageing but also in neurodevelopmental disorders, Dr. Dierssen concludes.

The study is published in the Journal of Cellular and Molecular Medicine.

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HIV drug may improve cognitive function in people with Down syndrome - Study Finds

Meet the company trying precision medicine for autism | Spectrum – Spectrum

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Ibudilast improved cognition and behavior in men with fragile X syndrome in a small clinical trial. The use of the other drug in STP1, bumetanide, in autistic people is cloudier. A 2020 clinical trial found that only some of the children who received bumetanide for three months showed reduced repetitive behaviors, though studies over the past two years have suggested that patterns of electrical activity in the brain or levels of specific immune molecules in the blood can predict a persons response to the drug. Last year, however, phase 3 studies of bumetanide were ended after researchers found no benefit to treatment.

Bumetanide is an excellent drug for autism, provided you select specific subpopulations of young people, says Yehezkel Ben-Ari, president and co-founder of French biotech company Neurochlore, which owns the patent for bumetanide as an autism treatment. Adults, he adds, may not respond as well. The 2020 trial found that younger children showed more improvement in social communication and responsiveness than did older ones.

Many people have hoped for precision medicine, but generally, the field of autism has moved away to some degree. Catherine Lord

Beyond the questions around bumetanide, others doubt that personalized therapy can be applied to autism at all. Many people have hoped for precision medicine, but generally, the field of autism has moved away to some degree, says Catherine Lord, distinguished professor of psychiatry at the University of California, Los Angeles. Thats because, she says, for something as complex and heterogeneous as autism, theres no clear link between known genetic factors and autism traits. Researchers have not yet had success in finding biomarkers for diagnosis of the condition, let alone predicting who is most likely to respond to treatment, she says.

Understanding the more convergent mechanisms of autism, and what is common about people with autism rather than whats different about them, is a more important approach to understanding whats treatable or preventable in the condition, says John Constantino, professor of psychiatry and pediatrics at Washington University in St. Louis, Missouri.

A molecular footprint found only in a subgroup of people with autism may have nothing to do with the condition and instead be related to other factors, such as depression or anxiety, Constantino says. So far, he adds, researchers havent even been able to reliably differentiate people with autism from those without the condition by looking at biological traits. Identifying a biological profile for different autism subtypes would be a significant advance in the field, he says, if Stalicla has actually done it.

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Meet the company trying precision medicine for autism | Spectrum - Spectrum

Ambys Medicines Announces Formation of Clinical and Scientific Advisory Boards with Leading Liver Disease and Cell and Gene Therapy Experts – Business…

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SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Ambys Medicines, a company pioneering cell-replacement therapies for patients with liver disease, today announced the formation of its clinical and scientific advisory boards comprising leading clinical experts in liver disease and hepatocyte transplantation, and world-class scientists pioneering cell and gene technologies.

The clinical advisory board provides guidance on advancing Ambyss lead program, AMI-918, through the clinic and provides critical input on clinical trial design and patient selection. The scientific advisory board will provide a cross-discipline perspective on applying cutting-edge technology to Ambyss cell therapy platform to progress its discovery pipeline of next generation cell therapies.

Were honored to work with such an esteemed and diverse group of experts in liver disease, hepatocyte transplantation, and cell and gene therapies whose collective experience will be highly valuable as we finalize our clinical development strategy for AMI-918 and progress our genetically engineered hepatocyte follow-on programs, said Ronald Park, M.D., Chief Executive Officer of Ambys Medicines. Were grateful for the engagement and support from our advisors as we work to bring first-in-class hepatocyte replacement therapies to liver failure patients who currently lack treatment options.

Each of our advisors brings incredible knowledge and expertise in their respective fields that will be instrumental to Ambys as we continue to broaden our pipeline and move closer to becoming a clinical-stage company, said Markus Grompe, M.D., Founder and Chief Scientific Officer of Ambys Medicines. Were excited to partner together to realize the potential of our novel replacement cell therapy platform in restoring lost hepatic function to patients with acute or chronic liver failure and genetic liver diseases.

Clinical Advisory Board

Scientific Advisory Board

About Ambys MedicinesAmbys Medicines is focused on pioneering cell replacement therapies for patients with liver failure. Ambyss proprietary platform enables the company to be the first and only company able to develop and manufacture functional human hepatocytes at scale. Our scientific approach has the potential to fundamentally transform the treatment paradigm for patients with acute and chronic liver failure and genetic diseases of the liver. Our lead program, AMI-918, is a hepatocyte replacement cell therapy in development to restore lost hepatic function. Beyond AMI-918, we are building a pipeline of next-generation modified hepatocytes that will rapidly expand the range of treatable patient populations. Learn more at ambys.com and follow us on Twitter, LinkedIn and Instagram.

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Ambys Medicines Announces Formation of Clinical and Scientific Advisory Boards with Leading Liver Disease and Cell and Gene Therapy Experts - Business...

CRISPR, 10 Years On: Learning to Rewrite the Code of Life – The New York Times

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Ten years ago this week, Jennifer Doudna and her colleagues published the results of a test-tube experiment on bacterial genes. When the study came out in the journal Science on June 28, 2012, it did not make headline news. In fact, over the next few weeks, it did not make any news at all.

Looking back, Dr. Doudna wondered if the oversight had something to do with the wonky title she and her colleagues had chosen for the study: A Programmable Dual RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity.

I suppose if I were writing the paper today, I would have chosen a different title, Dr. Doudna, a biochemist at the University of California, Berkeley, said in an interview.

Far from an esoteric finding, the discovery pointed to a new method for editing DNA, one that might even make it possible to change human genes.

I remember thinking very clearly, when we publish this paper, its like firing the starting gun at a race, she said.

In just a decade, CRISPR has become one of the most celebrated inventions in modern biology. It is swiftly changing how medical researchers study diseases: Cancer biologists are using the method to discover hidden vulnerabilities of tumor cells. Doctors are using CRISPR to edit genes that cause hereditary diseases.

The era of human gene editing isnt coming, said David Liu, a biologist at Harvard University. Its here.

But CRISPRs influence extends far beyond medicine. Evolutionary biologists are using the technology to study Neanderthal brains and to investigate how our ape ancestors lost their tails. Plant biologists have edited seeds to produce crops with new vitamins or with the ability to withstand diseases. Some of them may reach supermarket shelves in the next few years.

CRISPR has had such a quick impact that Dr. Doudna and her collaborator, Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens in Berlin, won the 2020 Nobel Prize for chemistry. The award committee hailed their 2012 study as an epoch-making experiment.

Dr. Doudna recognized early on that CRISPR would pose a number of thorny ethical questions, and after a decade of its development, those questions are more urgent than ever.

Will the coming wave of CRISPR-altered crops feed the world and help poor farmers or only enrich agribusiness giants that invest in the technology? Will CRISPR-based medicine improve health for vulnerable people across the world, or come with a million-dollar price tag?

The most profound ethical question about CRISPR is how future generations might use the technology to alter human embryos. This notion was simply a thought experiment until 2018, when He Jiankui, a biophysicist in China, edited a gene in human embryos to confer resistance to H.I.V. Three of the modified embryos were implanted in women in the Chinese city of Shenzen.

In 2019, a court sentenced Dr. He to prison for illegal medical practices. MIT Technology Review reported in April that he had recently been released. Little is known about the health of the three children, who are now toddlers.

Scientists dont know of anyone else who has followed Dr. Hes example yet. But as CRISPR continues to improve, editing human embryos may eventually become a safe and effective treatment for a variety of diseases.

Will it then become acceptable, or even routine, to repair disease-causing genes in an embryo in the lab? What if parents wanted to insert traits that they found more desirable like those related to height, eye color or intelligence?

Franoise Baylis, a bioethicist at Dalhousie University in Nova Scotia, worries that the public is still not ready to grapple with such questions.

Im skeptical about the depth of understanding about whats at issue there, she said. Theres a difference between making people better and making better people.

Dr. Doudna and Dr. Charpentier did not invent their gene-editing method from scratch. They borrowed their molecular tools from bacteria.

In the 1980s, microbiologists discovered puzzling stretches of DNA in bacteria, later called Clustered Regularly Interspaced Short Palindromic Repeats. Further research revealed that bacteria used these CRISPR sequences as weapons against invading viruses.

The bacteria turned these sequences into genetic material, called RNA, that could stick precisely to a short stretch of an invading viruss genes. These RNA molecules carry proteins with them that act like molecular scissors, slicing the viral genes and halting the infection.

As Dr. Doudna and Dr. Charpentier investigated CRISPR, they realized that the system might allow them to cut a sequence of DNA of their own choosing. All they needed to do was make a matching piece of RNA.

To test this revolutionary idea, they created a batch of identical pieces of DNA. They then crafted another batch of RNA molecules, programming all of them to home in on the same spot on the DNA. Finally, they mixed the DNA, the RNA and molecular scissors together in test tubes. They discovered that many of the DNA molecules had been cut at precisely the right spot.

For months Dr. Doudna oversaw a series of round-the-clock experiments to see if CRISPR might work not only in a test tube, but also in living cells. She pushed her team hard, suspecting that many other scientists were also on the chase. That hunch soon proved correct.

In January 2013, five teams of scientists published studies in which they successfully used CRISPR in living animal or human cells. Dr. Doudna did not win that race; the first two published papers came from two labs in Cambridge, Mass. one at the Broad Institute of M.I.T. and Harvard, and the other at Harvard.

Lukas Dow, a cancer biologist at Weill Cornell Medicine, vividly remembers learning about CRISPRs potential. Reading the papers, it looked amazing, he recalled.

Dr. Dow and his colleagues soon found that the method reliably snipped out pieces of DNA in human cancer cells.

It became a verb to drop, Dr. Dow said. A lot of people would say, Did you CRISPR that?

Cancer biologists began systematically altering every gene in cancer cells to see which ones mattered to the disease. Researchers at KSQ Therapeutics, also in Cambridge, used CRISPR to discover a gene that is essential for the growth of certain tumors, for example, and last year, they began a clinical trial of a drug that blocks the gene.

Caribou Biosciences, co-founded by Dr. Doudna, and CRISPR Therapeutics, co-founded by Dr. Charpentier, are both running clinical trials for CRISPR treatments that fight cancer in another way: by editing immune cells to more aggressively attack tumors.

Those companies and several others are also using CRISPR to try to reverse hereditary diseases. On June 12, researchers from CRISPR Therapeutics and Vertex, a Boston-based biotech firm, presented at a scientific meeting new results from their clinical trial involving 75 volunteers who had sickle-cell anemia or beta thalassemia. These diseases impair hemoglobin, a protein in red blood cells that carries oxygen.

The researchers took advantage of the fact that humans have more than one hemoglobin gene. One copy, called fetal hemoglobin, is typically active only in fetuses, shutting down within a few months after birth.

The researchers extracted immature blood cells from the bone marrow of the volunteers. They then used CRISPR to snip out the switch that would typically turn off the fetal hemoglobin gene. When the edited cells were returned to patients, they could develop into red blood cells rife with hemoglobin.

Speaking at a hematology conference, the researchers reported that out of 44 treated patients with beta thalassemia, 42 no longer needed regular blood transfusions. None of the 31 sickle cell patients experienced painful drops in oxygen that would have normally sent them to the hospital.

CRISPR Therapeutics and Vertex expect to ask government regulators by the end of year to approve the treatment.

Other companies are injecting CRISPR molecules directly into the body. Intellia Therapeutics, based in Cambridge and also co-founded by Dr. Doudna, has teamed up with Regeneron, based in Westchester County, N.Y., to begin a clinical trial to treat transthyretin amyloidosis, a rare disease in which a damaged liver protein becomes lethal as it builds up in the blood.

Doctors injected CRISPR molecules into the volunteers livers to shut down the defective gene. Speaking at a scientific conference last Friday, Intellia researchers reported that a single dose of the treatment produced a significant drop in the protein level in volunteers blood for as long as a year thus far.

The same technology that allows medical researchers to tinker with human cells is letting agricultural scientists alter crop genes. When the first wave of CRISPR studies came out, Catherine Feuillet, an expert on wheat, who was then at the French National Institute for Agricultural Research, immediately saw its potential for her own work.

I said, Oh my God, we have a tool, she said. We can put breeding on steroids.

At Inari Agriculture, a company in Cambridge, Dr. Feuillet is overseeing efforts to use CRISPR to make breeds of soybeans and other crops that use less water and fertilizer. Outside of the United States, British researchers have used CRISPR to breed a tomato that can produce vitamin D.

Kevin Pixley, a plant scientist at the International Maize and Wheat Improvement Center in Mexico City, said that CRISPR is important to plant breeding not only because its powerful, but because its relatively cheap. Even small labs can create disease-resistant cassavas or drought-resistant bananas, which could benefit poor nations but would not interest companies looking for hefty financial returns.

Because of CRISPRs use for so many different industries, its patent has been the subject of a long-running dispute. Groups led by the Broad Institute and the University of California both filed patents for the original version of gene editing based on CRISPR-Cas9 in living cells. The Broad Institute won a patent in 2014, and the University of California responded with a court challenge.

In February of this year, the U.S. Patent Trial and Appeal Board issued what is most likely the final word on this dispute. They ruled in favor of the Broad Institute.

Jacob Sherkow, an expert on biotech patents at the University of Illinois College of Law, predicted that companies that have licensed the CRISPR technology from the University of California will need to honor the Broad Institute patent.

The big-ticket CRISPR companies, the ones that are farthest along in clinical trials, are almost certainly going to need to write the Broad Institute a really big check, he said.

The original CRISPR system, known as CRISPR-Cas9, leaves plenty of room for improvement. The molecules are good at snipping out DNA, but theyre not as good at inserting new pieces in their place. Sometimes CRISPR-Cas9 misses its target, cutting DNA in the wrong place. And even when the molecules do their jobs correctly, cells can make mistakes as they repair the loose ends of DNA left behind.

A number of scientists have invented new versions of CRISPR that overcome some of these shortcomings. At Harvard, for example, Dr. Liu and his colleagues have used CRISPR to make a nick in one of DNAs two strands, rather than breaking them entirely. This process, known as base editing, lets them precisely change a single genetic letter of DNA with much less risk of genetic damage.

Dr. Liu has co-founded a company called Beam Therapeutics to create base-editing drugs. Later this year, the company will test its first drug on people with sickle cell anemia.

Dr. Liu and his colleagues have also attached CRISPR molecules to a protein that viruses use to insert their genes into their hosts DNA. This new method, called prime editing, could enable CRISPR to alter longer stretches of genetic material.

Prime editors are kind of like DNA word processors, Dr. Liu said. They actually perform a search and replace function on DNA.

Rodolphe Barrangou, a CRISPR expert at North Carolina State University and a founder of Intellia Therapeutics, predicted that prime editing would eventually become a part of the standard CRISPR toolbox. But for now, he said, the technique was still too complex to become widely used. Its not quite ready for prime time, pun intended, he said.

Advances like prime editing didnt yet exist in 2018, when Dr. He set out to edit human embryos in Shenzen. He used the standard CRISPR-Cas9 system that Dr. Doudna and others had developed years before.

Dr. He hoped to endow babies with resistance to H.I.V. by snipping a piece of a gene called CCR5 from the DNA of embryos. People who naturally carry the same mutation rarely get infected by H.I.V.

In November 2018, Dr. He announced that a pair of twin girlshad been born with his gene edits. The announcement took many scientists like Dr. Doudna by surprise, and they roundly condemned him for putting the health of the babies in jeopardy with untested procedures.

Dr. Baylis of Dalhousie University criticized Dr. He for the way he reportedly presented the procedure to the parents, downplaying the radical experiment they were about to undertake. You could not get an informed consent, unless you were saying, This is pie in the sky. Nobodys ever done it, she said.

In the nearly four years since Dr. Hes announcement, scientists have continued to use CRISPR on human embryos. But they have studied embryos only when theyre tiny clumps of cells to find clues about the earliest stages of development. These studies could potentially lead to new treatments for infertility.

Bieke Bekaert, a graduate student in reproductive biology at Ghent University in Belgium, said that CRISPR remains challenging to use in human embryos. Breaking DNA in these cells can lead to drastic rearrangements in the chromosomes. Its more difficult than we thought, said Ms. Bekaert, the lead author of a recent review of the subject. We dont really know what is happening.

Still, Ms. Bekaert held out hope that prime editing and other improvements on CRISPR could allow scientists to make reliably precise changes to human embryos. Five years is way too early, but I think in my lifetime it may happen, she said.

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CRISPR, 10 Years On: Learning to Rewrite the Code of Life - The New York Times

A Functional Medicine Approach to Detoxing the Body – Magazine of Santa Clarita

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Daily our bodies are exposed to toxic chemicals from the environment that can be harmful to our health. Exposure to these toxins comes in many forms, including the air we breathe, the stress we feel, and the food we eat. Most of the harmful toxins are man-made artificial products introduced into the environment due to human activity. Examples of toxins include industrial waste products, pesticides, herbicides, food additives, and preservatives. Several metals (arsenic, lead, mercury, cadmium, and PCBs) are also believed to cause disease.Once those harmful chemicals enter the body they are referred to as xenobiotics. Xenobiotics is a chemical compound (drug, pesticide, or carcinogen) that is foreign to the human body. Several of these xenobiotics are endocrine disruptors, meaning that they mimic or interfere with your bodys hormones. For example, arsenic can poison the beta-cell responsible for producing insulin in the pancreas and damage our DNA. It is suggested that arsenic may cause 15%-20% of diabetes.The body has a natural detoxification system through the work of the liver, kidneys, large intestines, lymphatic system, and sweat glands. The liver is the major player in the bodys detox system, but they all work together to reduce the buildup of toxins. We all have different toxin exposure, and we all have genetic differences in how a persons body is able to produce the enzymes that regulate detoxification. When a persons body burden of toxins has exceeded the bodys ability to eliminate them, symptoms can occur. A few of the most common symptoms include fatigue, headaches, anxiety, depression, brain fog, difficulty losing weight, skin breakouts, and digestive issues.Lowering your toxic load is vital to your health. However, what we eat can either support or hinder the bodys ability to process and eliminate harmful toxins. Several nutrients are needed to push detox pathways in the body and support and regulate detoxification. A deficiency in any of them could cause an increased body burden or buildup of chemicals in the body.In functional medicine, we take a whole-body approach to toxin load by checking for nutritional status, genetic susceptibility to nutritional deficiencies, and environmental exposures. It is important to investigate exposure pathways to identify a source of toxicity elevation.One of the best ways to protect yourself is to identify the toxic exposure and stop it! It is important to support the bodys natural detoxification system by eating organic colorful, plant-based diet rich in antioxidants. Finally, promote glutathione (powerful antioxidant) production in the body by taking vitamin C, eating sulfur-rich foods, and getting restorative sleep as it may increase the excretion of toxins. For more information, call Compassionate Healthcare Associates, 661 295-7777.

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A Functional Medicine Approach to Detoxing the Body - Magazine of Santa Clarita

After 9 Years in Trials, Roche’s Crenezumab Stalls Out – Being Patient

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By Simon Spichak, MSc | June 28th, 2022

There were more than 140 experimental Alzheimers drugs in clinical trials as of the start of 2022. On the one hand, this number inspires some optimism that something will break through. But at the same time, each of these drugs faces more than a 99-percent chance of failure. This month, another potential disease-modifying Alzheimers drug, which has been in trials for nearly a decade, is dropping out of the race.

Genentech, a biotech company under Roche, announced that their anti-amyloid drug crenezumab, developed in conjunction with Switzerland-based biotech company AC Immune SA, has ultimately failed to slow or prevent Alzheimers disease in a cohort of cognitively healthy people with early-onset Alzheimers genes.

Were disappointed that the treatment did not demonstrate a statistically significant clinical benefit, Dr. Eric M. Reiman, Banner Alzheimers Institute executive director and one of the study leaders, said in a news release. At the same time, were proud of the impact that this precedent-setting trial has had in shaping a new era in Alzheimers prevention research and were extremely grateful to our research participants and their families.

In the Phase 2 clinical trial, 252 people were randomized to receive crenezumab or placebo over the course of five to eight years. But the drug failed to prevent the decline in cognitive function or episodic memory which occurs as a result of early-onset Alzheimers.

The participants in the clinical trial had autosomal dominant Alzheimers disease which is caused by mutations in the APP, PSEN1 or PSEN2 genes. Even carrying one copy of these genes leads to greater build up of amyloid plaques and symptoms developing in people between their 30s and 60s.

While this attempt at targeting beta-amyloid plaques failed to improve cognitive outcomes, it is unclear whether the drug effectively reduced plaque build-up, highlighting just how little the research community knows for certain about how Alzheimers forms and progresses.

David Knopman, a professor at the Mayo Clinic College of Medicine wrote that crenezumab previously failed Phase 3 clinical trials for non-genetic forms of Alzheimers disease, even though the drug was administered at a higher dose than in this recent study.

Knopman added, The crenezumab trial in the Colombian dominantly inherited [Alzheimers] cohort is disappointing, but not altogether unpredicted based on earlier failures of crenezumab in sporadic Alzheimers, and based on the inability of crenezumab to clear brain amyloid to any extent.

Marc Aurel Busche and Samuel Harris, both physician-scientists at University College London, emphasized that we know very little about the role of amyloid proteins and beta-amyloid plaques in the healthy brain. In one study authored by Busche, anti-amyloid antibodies actually worsened Alzheimers pathology in mice.

We conclude that our understanding of the effect of anti-amyloid antibodies on brain (dys)function in humans with [Alzheimers] is rudimentary, they wrote, adding that therapeutic benefits could be masked by detrimental effects of anti-amyloid antibodies.

The first-ever FDA-approved disease-modifying drug for Alzheimers, Aduhelm (aducanumab) was designed with similar mechanisms. Another monoclonal antibody, Aduhelm targets beta-amyloid, too. But, since its FDA approval, the drug has been mired in controversy over unclear efficacy data. While some researchers and drug developers are exploring other avenues to addressing Alzheimers altogether from the microbiome to tau protein tangles the research community hasnt given up on beta-amyloid as a target, nor on these -mab drugs as an approach.

In the meantime, Roche is focused on advancing its other anti-amyloid drug, gantenerumab. This anti-amyloid received a Breakthrough Therapy designation from the Food and Drug Administration in October 2021.

Gantenerumab failed Phase 3 clinical trial in 2014 but was brought back as an Alzheimers drug candidate in 2017, to test whether it may provide preventative benefits. The ongoing Phase 3 trial is exploring whether the drug can prevent cognitive decline in healthy individuals aged 60 to 80 who have evidence of Alzheimers biomarker beta-amyloid build-up in the brain.

Unlike other anti-amyloid drugs, i.e. Aduhelm, gantenerumab is not administered intravenously. Instead, it would be delivered via subcutaneous injection where the drug is administered under the skin potentially enabling at-home treatment. Results from a Phase 3 clinical trial assessing its efficacy in early Alzheimers is expected by the end of the year.

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After 9 Years in Trials, Roche's Crenezumab Stalls Out - Being Patient

Generation Bio Reports Business Highlights and First Quarter 2022 Financial Results – GuruFocus.com

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Company continues to optimize cell-targeted lipid nanoparticle (ctLNP) delivery system for nonviral genetic medicine applications in liver, retina and vaccines

Cash balance of $337.0M expected to fund operations into 2024

CAMBRIDGE, Mass., May 05, 2022 (GLOBE NEWSWIRE) -- Generation Bio Co. ( GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and first quarter 2022 financial results.

We are making steady advances in optimizing our cell-targeted lipid nanoparticles, or ctLNPs, for use in our liver and retina programs as well as in developing our platform technologies for novel vaccine applications, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. We believe our approach to genetic medicine has the potential to create highly differentiated therapies capable of fulfilling significant unmet need, and we look forward to providing updates on our progress.

Key priorities across core therapeutic areas:

First Quarter 2022 Financial Results

About Generation Bio

Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral genetic medicine platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific cell types, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bios manufacturing scale to hundreds of millions of doses to support its mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.

For more information, please visit http://www.generationbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platform, our research and clinical development plans, applications and preclinical data and other statements containing the words believes, anticipates, plans, expects, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from earlier preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties regarding the timing and ability to complete the build-out of the companys manufacturing facility and regarding the RES manufacturing process; challenges in the manufacture of genetic medicine products; whether the companys cash resources are sufficient to fund the companys operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the companys business and operations; expectations for regulatory approvals to conduct trials or to market products; as well as the other risks and uncertainties set forth in the Risk Factors section of our most recent annual report on Form 10-K, which is on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the companys views as of the date hereof. The company anticipates that subsequent events and developments will cause the companys views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the companys views as of any date subsequent to the date on which they were made.

Investors and Media ContactMaren KillackeyGeneration Bio857-371-4638[emailprotected]

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited)(in thousands, except share and per share data)

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Generation Bio Reports Business Highlights and First Quarter 2022 Financial Results - GuruFocus.com

Dr. Stephen Kingsmore to receive Luminary Award at 2022 Precision Medicine World Conference – EurekAlert

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image:Stephen Kingsmore, MD, DSc, President and CEO or Rady Children's Institute for Genomic Medicine view more

Credit: Rady Children's Institute for Genomic Medicine

SAN DIEGO, Calif. June 27, 2022 Rady Children's Institute for Genomic Medicine (RCIGM) today announced that Stephen Kingsmore, MD, DSc, President and CEO, will receive the 2022 Luminary Award at the Precision Medicine World Conference (PMWC) to be held from June 28 to 30 in the Silicon Valley region of California.

The Luminary Award recognizes the recent contributions of prominent figures who have accelerated precision medicine into the clinic. Dr. Kingsmore will be recognized for his innovation in rapid neonatal molecular diagnoses using whole-genome sequencing. Additional PMWC 2022 honorees will include Dr. Albert Bourla from Pfizer and Dr. Stephen Hoge from Moderna.

RCIGM at PMWC:

On Wednesday, June 29, at 9 a.m., PST, Dr. Kingsmore will received the Luminary Award in a brief presentation by Martin Reese Ph.D., Co-Founder, CEO, Pres., Fabric Genomics.

Then at 9:15 a.m., PST, Dr. Kingsmore will speak on NICU Current State and Future of Whole Genome Sequencing as part of the Track 4 program, Sequencing in the Neonatal and Pediatric Intensive Care Unit. Dr. Kingsmore will be present to receive his award at this time.

Later at 11:15 a.m. PST, Russell Nofsinger, PhD, Senior Director of Business Development at RCIGM, will present on Partnerships to End the Diagnostic and Therapeutic Odysseys during the Clinical DX Showcase.

RCIGM will also host Booth #C1626 Hall C, Track 3, from June 28-30 during regular conference hours.

Rady Childrens Institute for Genomic Medicine

Rady Childrens Institute for Genomic Medicine is transforming neonatal and pediatric health care by harnessing the power of Rapid Precision Medicine to improve the lives of children and families facing rare genetic disease. Founded by Rady Childrens Hospital and Health Center, the Institute offers the fastest delivery of rapid Whole Genome Sequencing to enable prompt diagnosis and targeted treatment of critically ill newborns and children in intensive care. The Institute now provides clinical genomic diagnostic services for a growing network of more than 70 childrens hospitals. The vision is for this life-changing technology to become standard of care and enable clinicians nationwide to provide rapid, personalized care. Learn more about the non-profit Institute at RadyGenomics.org. Follow us on Twitter and LinkedIn.

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Dr. Stephen Kingsmore to receive Luminary Award at 2022 Precision Medicine World Conference - EurekAlert

Do agri-businesses ‘control’ agriculture? The emerging gene-editing revolution in Latin America is challenging that belief – Genetic Literacy Project

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Small actions can create such huge differences. We see that butterfly effect playing out in Latin America. where the development of a new generation of crops using CRISPR and other new breeding techniques (NBTs) is now unfolding and whats smaller than a gene?

Changes in a single gene are revolutionizing science, making a big impact on medicine and agriculture: think COVID vaccines or genes that increase yield or climate resilience or nutritional value of crops. In the food sector, gene-edited tomatoes, wheat and other foods are already being consumed and commercialized, and dozens more are on the way.

The main challenge going forward in the agriculture sector, particularly in Latin America, is how NBTs will be regulated. As GMOs in which genes are moved between species to introduce specific traits? Transgenic techniques are heavily regulated in most jurisdictions because of the presence of so-called foreign genes.

Many of the innovation-blocking regulations were composed in the early 2000s when much less was known about genes, and fear and misunderstanding drove public opinion against the science, and politicians followed suit.

Scientists believe the obsession of distinguishing between what is natural and what is a foreign gene is scientifically silly, as even in nature genes flow from one species to another. This dubious notion has long throttled the biotech ag revolution.

If this is the regulatory model going forward, the CRISPR ag revolution will stall. Gene editingwhich is known as cisgenicscan introduce many new traits but is not a substitute in many cases for transgenicsdoes not involve moving genes among species.

The EU, at least for now, seems intent on sticking to the backward GMO regulations it adopted in 2001 and 2003 in the wake of food scares unrelated to biotechnology. The United States, Canada, China, Japan, and even the United Kingdom are shedding decade-old superstitions and taking steps to greenlight gene editing.

Latin American nations are also taking global leadership in the research and development of gene-edited crops. Argentina, Brazil, and other South American countries are being proactive, creating regulatory frameworks that will make these novel crops accessible to farmers and consumers in just a few years time. They will be evaluated as conventional crops, making the research and development process needed for environmental release and testing affordable to developers something that does not happen with transgenic GMOs in which only big companies can afford to run the expensive, time-consuming regulatory gauntlet.

The relatively minuscule costs necessary to bring new products to the evaluation stage could help unleash an entrepreneurial revolution in the food sector, ironically undercutting the influence of multinational agribusinesses. This regulatory revolution could reshape the publics perception of genetically modified crops and knee-cap aggressive anti-biotech activist groups that have spent decades spreading alarmist, unfounded fears that multinational corporations are out to control the global food supply.

In Latin America, agricultural biotechnology public research centers are popping up, allocating resources to develop novel crop varieties. New and small companies are being founded and funded. Together they are revolutionizing the future of food production in Latin America, with crops and foods being developed from a farmers and consumers perspective and by creating novel crop varieties with regional interest.

In Argentina, BioHeuris, an expert in weed management, is leading the synthetic biology revolution, working with CRISPR tools to develop next-generation herbicide-resistant soybean, sorghum, alfalfa, rice, and cotton. Created in 2016, BioHeuris accelerates plant breeding to evolve crops faster than weeds. Together with integrated pest management techniques, they expect to develop a sustainable system for numerous crops with varieties available for commercialization by 2026 or 2027.

With our platforms fully operational, time to market will be reduced by half and development costs cut 100 times compared to current GMO crops developed by the big seed companies, said co-founder and Director of Strategy Carlos Perez.

CONABIA, Argentinas biosafety commission, has already ruled that these gene-edited plants should not be regulated as GMOs. In January, APHIS in the United States and Brazils CTNBio (National Technical Biosafety Commission) also gave the green light for the introduction of BioHeuris CRISPR solution.

In Brazil, the government-funded company Embrapa is emerging as an innovation leader in exploiting. It developed the first gene-edited sugarcane in the world. It has two varieties: one offers higher cell wall digestibility and a higher concentration of sucrose in plant tissues. CTNBio has determined that neither variety will be regulated as a transgenic GMO.

According to the deputy head of Research and Development at Embrapa, the development of new cultivars through CRISPR is at the frontier of knowledge.

These cultivars are only the beginning, and they pave the way for the development and delivery of other cultivars for the production sector with characteristics that will directly impact the productivity and reduce production costs.

Colombia is following a different path. CIAT (International Center for Tropical Agriculture), a public research center, has been identified as a pioneer in genome editing in Colombia and is leading the national research and development of gene-edited crops. CIATs researchers have worked on several crops including cassava, rice, and beans. The modified rice variety is resistant to the hoja blanca virus, a disease that bleaches the leaves off and eventually kills rice plants. It is prevalent in Latin America. CIATs team is also exploring the use of genome editing to make it easier for people to digest beans.

Chiles research team is based in the Department of Biology of Sciences Faculty at the University of Chile. It is working on creating gene-edited crops, focusing on tomatoes and kiwi that can tolerate drought and soil salinity. In addition, they are using CRISPR to modify apples to increase their nutritional profile, adding a higher content of carotenoids, and resisting the oxidation that causes browning after they are cut.

In addition to the efforts made by the University of Chile, NeoCrop, an emerging company, is joining them in the domestic research landscape of gene-edited crops. They are developing gene-edited wheat that produces higher levels of fiber and is resistant to drought and extreme heat. NeoCrop expects to have this new gene-edited wheat variety ready for field trials this year or next.

This emerging dynamic offers a preview of the future in which small and entrepreneurial start-ups will lead in the research and development of novel crops with a focus on farmers and consumers. Increasingly, NGO criticism that crop biotechnology in Latin America is being driven by monopolistic corporations is obsolete and misguided.

Luis Ventura is a trained biologist and biosafety expert. Luis is a member of Mexican Scientist Allies for Knowledge in Agriculture and a 2016 Alliance for Science Global Leadership Fellow and trainer. View Luis LinkedIn pagehere. Find Luis on Twitter@Iuisventura

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Do agri-businesses 'control' agriculture? The emerging gene-editing revolution in Latin America is challenging that belief - Genetic Literacy Project

Akouos Presents Nonclinical Data Supporting the Planned Clinical Development of AK-OTOF and Strategies for Regulated Gene Expression in the Inner Ear…

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- Nonclinical data demonstrate that a single intracochlear administration of an AAVAnc80 vector led to durable restoration of auditory function and was well tolerated, supporting planned clinical development of AK-OTOF for the treatment of OTOF-mediated hearing loss

- MicroRNA target site (miR-TS)-incorporation in AAV vectors is shown to have potential benefits for de-targeting transgene expression in the inner ear, supporting future development of gene therapies targeting a broad range of inner ear conditions

BOSTON, May 19, 2022 (GLOBE NEWSWIRE) -- Akouos, Inc. ( AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, presented nonclinical data at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting. The company gave two nonclinical presentations at the meeting: one that supports the planned clinical development of AK-OTOF, a gene therapy intended for the treatment of OTOF-mediated hearing loss; and another that supports the potential use of microRNA target site (miR-TS) in adeno-associated viral (AAV) vectors for regulated gene expression in the inner ear.

We are excited to present these nonclinical data, which highlight our precision genetic medicine platform and the potential of genetic medicines to address a broad range of inner ear conditions, to the gene and cell therapy community. The AK-OTOF nonclinical data demonstrate durable restoration of auditory function and show that the product candidate was systemically and locally well tolerated in two translationally relevant animal species, said Manny Simons, Ph.D., founder, president, and chief executive officer of Akouos. As we continue to progress toward planned IND submissions for AK-OTOF in the first half of 2022 and AK-antiVEGF in 2022, we are encouraged by the growing body of evidence supporting these filings, as well as by our efforts to advance preclinical development of other potential gene therapies for inner ear conditions, such as GJB2-mediated hearing loss, and to develop platform capabilities that can be applied to regenerative medicine approaches in the inner ear.

Nonclinical In Vivo Expression, Durability of Effect, Biodistribution/Shedding, and Safety Evaluations Support Clinical Development of AK-OTOF (AAVAnc80-hOTOF Vector) for OTOF-mediated Hearing Loss Presenting Author: Ann E. Hickox, Ph.D.Session Title and Room: Ophthalmic and Auditory Diseases; Salon G

AK-OTOF is an AAV vector-based gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss by delivering transgenes encoding OTOF to inner hair cells (IHCs). Following intracochlear delivery, and subsequent co-transduction of IHCs by each component vector, the two transgene products recombine to generate a full-length otoferlin mRNA transcript and subsequently a full-length otoferlin protein. Results from this presentation show:

Together, these nonclinical studies further support the planned clinical development of AK-OTOF for the treatment of OTOF-mediated hearing loss.

The digital presentation is located at https://akouos.com/gene-therapy-resources/.

Evaluating miR-Target Sites as a Strategy to Allow AAV Vector-based De-targeting of Gene Expression in the Inner EarPresenting Author: Richard M. Churchill Jr.Poster Board Number: Tu-37

In the development of AAV gene therapy vectors, a goal is to generate safe and effective product candidates that deliver targeted transgene expression. Ubiquitous promoters can drive strong widespread expression in the inner ear in mice and NHPs. This expression can be well tolerated across the inner ear, as is the case for Akouoss first two programs, AK-OTOF and AK-antiVEGF. Addition of selective cis-regulatory elements may be needed for some transgenes, such as GJB2, where expression in a portion of nontarget cells is not well tolerated. This nonclinical study explored the potential use of miR-TS incorporation in AAV vectors for de-targeting transgene expression in different cell types of the cochlea. Using an in vitro model, expression of transgene mRNA and protein in the presence or absence of the target sites was evaluated. Akouos identified multiple microRNA target sites to drive various differential expression patterns demonstrating that a combination of AAVAnc80 and miR-TS can drive expression in supporting cells, while limiting expression in hair cells in cochlear explants. Future work will focus on evaluating miR-TS regulation in vivo and identifying combinations of different miR-TSs to enhance de-targeting in specific cell types where, for example, expression driven by ubiquitous promoters is not well tolerated.

The digital presentation is located at https://akouos.com/gene-therapy-resources/.

About AkouosAkouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Forward-Looking StatementsStatements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, and the timing of our IND submissions for AK-OTOF and AK-antiVEGF. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations and any changes in such laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors section of our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, which is on file with the Securities and Exchange Commission, and in other filings that Akouos may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts

Media:Katie Engleman, 1AB[emailprotected]

Investors:Courtney Turiano, Stern Investor Relations [emailprotected]

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Akouos Presents Nonclinical Data Supporting the Planned Clinical Development of AK-OTOF and Strategies for Regulated Gene Expression in the Inner Ear...

New hope for IVF patients as global study published in Human Reproduction shows AI can effectively assess genetic integrity of embryos USA – English -…

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SAN FRANCISCO, June 28, 2022 /PRNewswire/ -- Human Reproduction journal has published the ground-breaking results of an international clinical study, where a novel AI algorithm called Life Whisperer Genetics was developed by AI healthcare company Presegen to assess the genetic integrity of embryos using only images. The assessment is non-invasive, low-cost, and provides results instantly. This is in stark contrast with PGT-A, the standard method used in IVF today, which requires an invasive and potentially risky biopsy to remove a portion of the embryo, followed by an expensive and time-consuming genetic testing procedure.

The study was conducted with IVF clinics globally, including Ovation Fertility (USA), IVF-Life (Europe), Alpha IVF & Women's Specialists (SE Asia), and Wings IVF (India). Results showed that the AI algorithm could identify whether an embryo was genetically normal, or 'euploid'. Identification of euploid embryos can result in improved clinical outcomes, such as a better chance at pregnancy success.

Presagen's Chief Medical Science Officer Dr Sonya Diakiw explained "Because this assessment is based on images alone, it is not as accurate as PGT-A itself, which involves actual DNA sequencing. But we are finding that PGT-A results themselves can be variable, as they depend on the embryo sample being tested. PGT-A only tests 5 cells from a total of around 200, so it is not always representative of the entire embryo. Life Whisperer Genetics is a whole-embryo assessment of genetic integrity that does not require any invasive procedures, which can be used to prioritize embryos for use in IVF procedures."

The technology was evaluated prospectively on patients in Europe in collaboration with the IVF-Life Group. Dr Jon Aizpurua from IVF-Life said "Life Whisperer Genetics can be used for patients as a pre-screen, to ensure we only genetically test embryos that are likely to be normal, saving patients time and money. For patients who are not comfortable with invasive genetic tests, or in countries like Germany where invasive genetic tests are not permitted, Life Whisperer Genetics is a viable alternative to help select embryos that are most likely to be euploid."

Prospective studies were also performed in collaboration with Alpha IVF & Women's Specialists in Malaysia. Chief Embryologist Adelle Yun Xin Lim said "Computer vision with AI may revolutionise IVF treatment and this new technique is another milestone of AI in IVF. The technique will help doctors and embryologists around the world to predict the chromosome status of embryos in a rapid and non-invasive manner enabling the prioritization of embryos that are likely to be euploid for transfer or for further confirmatory PGT testing, leading to a faster time to pregnancy and reducing the cost of the treatment."

Ovation Fertility's VP of Scientific Advancement, Dr Matthew (Tex) VerMilyea said "This new product is very exciting. In some ways it is like a 'Rapid Antigen Test (RAT)' for embryo assessment, providing a non-invasive, instantaneous evaluation of genetic integrity, which will have massive potential for the US market when it receives FDA approval."

Life Whisperer Genetics is already available for IVF clinics and their patients in over 40 countries globally. It can be used in combination with Life Whisperer Viability, which assesses if an embryo is likely to lead to a pregnancy. International clinical studies have shown that Life Whisperer Viability can perform better than embryologists' current manual embryo assessment methods. Together, Life Whisperer Viability and Life Whisperer Genetics provide a comprehensive assessment of embryo quality.

Paper Title

Development of an artificial intelligence model for predicting the likelihood of human embryo euploidy based on blastocyst images from multiple imaging systems during IVF

https://academic.oup.com/humrep/advance-article/doi/10.1093/humrep/deac131/6604228

Authors

S. M. Diakiw1, J. M. M. Hall1,2,3, M. D. VerMilyea4,5, J. Amin6, J. Aizpurua7, L. Giardini7, Y. G. Briones7, A. Y. X. Lim8, M. A. Dakka1, T. V. Nguyen1, D. Perugini1, M. Perugini1,9

Paper Abstract

STUDY QUESTION

Can an artificial intelligence (AI) model predict human embryo ploidy status using static images captured by optical light microscopy?

SUMMARY ANSWER

Results demonstrated predictive accuracy for embryo euploidy, and showed a significant correlation between AI score and euploidy rate, based on assessment of images of blastocysts at Day 5 after IVF.

MAIN RESULTS AND THE ROLE OF CHANCE

Overall accuracy for prediction of euploidy on a blind test dataset was 65.3%, with a sensitivity of 74.6%. When the blind test dataset was cleansed of poor quality and mislabeled images, overall accuracy increased to 77.4%. This performance may be relevant to clinical situations where confounding factors, such as variability in PGT-A testing, have been accounted for. There was a significant positive correlation between AI score and the proportion of euploid embryos, with very high scoring embryos (9.0-10.0) twice as likely to be euploid than the lowest scoring embryos (0.0-2.4). When using the genetics AI model to rank embryos in a cohort, the probability of the top-ranked embryo being euploid was 82.4%, which was 26.4% more effective than using random ranking, and ~13-19% more effective that using the Gardner score. The probability increased to 97.0% when considering the likelihood of one of the top two ranked embryos being euploid, and the probability of both top two ranked embryos being euploid was 66.4%. Additional analyses showed that the AI model generalized well to different patient demographics and could also be used for evaluation of Day 6 embryos and for images taken using multiple time-lapse systems. Results suggested that the AI model could potentially be used to differentiate mosaic embryos based on the level of mosaicism.

WIDER IMPLICATIONS OF THE FINDINGS

These findings collectively support the use of this genetics AI model for evaluation of embryo ploidy status in a clinical setting. Results can be used to aid in prioritizing and enriching for embryos that are likely to be euploid for multiple clinical purposes, including selection for transfer in the absence of alternative genetic testing methods, selection for cryopreservation for future use, or selection for further confirmatory PGT-A testing, as required.

About Presagen and Life Whisperer

Presagen is an AI healthcare company that is changing the way clinics, patients, and medical data from around the world are connected through AI. Its platform, The Social Network for Healthcare, connects clinics and patients globally, and enables collaboration and data sharing to create scalable AI healthcare products that are affordable and accessible for all. The decentralized network democratizes the creation of AI products, promotes collaboration through incentives, and protects data privacy and ownership. With a focus on improving Women's Health outcomes globally, Presagen's first product, Life Whisperer, is being used by IVF clinics globally to improve pregnancy outcomes for couples struggling with fertility. With a vision of creating the largest network of clinics, patients, and medical data from around the world, Presagen is driving the future of AI Enhanced Healthcare.

About Ovation Fertility

Ovation Fertility is a national network of reproductive endocrinologists and scientific thought leaders focused on reducing the cost of having a family through more efficient and effective fertility care. Ovation's IVF and genetics laboratories, along with affiliated physician practices, work collaboratively to raise the bar for IVF treatment, with state-of-the-art, evidence-based fertility services that give hopeful parents the best chance for a successful pregnancy. Physicians partner with Ovation to offer their patients advanced preconception carrier screening; preimplantation genetic testing; donor egg and surrogacy services; and secure storage for their frozen eggs, embryos and sperm. Ovation also helps IVF labs across America improve their quality and performance with expert off-site lab direction and consultation. Learn more about Ovation's vision of a world without infertility at http://www.OvationFertility.com.

About IVF-Life

IVF-Life is a group of fertility clinics specialized in complex cases. Centres located inSpain and the UK have the latest advances in Reproductive Medicine and outstanding professionals in this field. The constant innovation and a firm commitment to technology keep IVF-Life at the forefront in the assisted reproduction field treating patients from all over the world with ahigh degree of success. IVF-Life perform a comprehensive range of treatments and diagnostic tests with the aim to provide effective solutions to a wide variety of fertility problems.

About Alpha IVF & Women's Specialists

Alpha IVF group comprises IVF centres in Kuala Lumpur, Penang and Singapore. Alpha IVF is a world-class fertility treatment provider bringing the most advanced fertility technologies and excellent success rates in achieving the goal of having a baby. Alpha IVF consists of a team of highly qualified and skilled doctors, scientists and nurses that deliver international standards of patient care. As the name Alpha suggests, the team have pioneered numerous innovative fertility treatments. Alpha IVF offers its patients access to a network of fertility experts and facilities fully equipped with a full range of cutting-edge laboratories, innovative technologies such as Artificial Intelligence, Next Generation Sequencing (NGS), 100% post-warm survival rate for embryo cryopreservation, time-lapse embryo monitoring, PIEZO-ICSI, sperm separation technologies and many others. Continuous R&D have led Alpha IVF to achieve numerous world firsts and innovative fertility treatment protocols both regionally and globally.

About Wings

Wings IVF comprises a chain of leading fertility clinics across India, with more than 12,000 live births through IVF. Wings hospitals are state of art specialty hospitals & clinics providing all infertility treatments and IVF. They provide top quality, comprehensive, holistic care to women of India at a reasonable cost. An interdisciplinary team of expert and caring professionals is committed to meeting the physical as well as emotional and spiritual needs for each woman and her family. The Wings Hospitals have been designed and furnished to provide a high level of fertility care with comfort and privacy.

SOURCE Presagen

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New hope for IVF patients as global study published in Human Reproduction shows AI can effectively assess genetic integrity of embryos USA - English -...

Worthy of medicine, worthy of good care and worthy of love | Carle.org – Carle Health

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At the age 18 while hooked up to monitors in a hospital bed and believing he was going to die of a severe allergic reaction, Bernie Ranchero, MD, MBA, confessed to his pastor he struggled with his sexuality and that he was gay. Growing up in a conservative religious family during the 80s and90s while the AIDs epidemic was shaping public perceptions of homosexuality had affected how he saw himself and made him want to hide who he was. It would be many years after his deathbed confession for the physician to fully come to terms with his identity and to become accepting of his own self-worth.

When I was 31 years old, Dr. Ranchero said, I reconciled my faith and sexuality, and I came to the realization that I was not an abomination. I had to overcome my own bias I had toward myself.

Overcoming his personal struggles and hearing of his patients medical experiences in rural Illinois drives him to address the challenges those in the LGBTQIA+ community face. Dr. Ranchero a Carle Illinois College of Medicine clinical assistant professor, the Coles County Public Health Department medical director and a practicing family medicine physician presented Killing me softly. Discrimination and inequality in medicine in a recent education series on gender inclusive care.

Dr. Ranchero discussed how shame and fear of discrimination can be barriers to care among the LGBTQIA+ community, especially for those in areas lacking diversity. Being different can mean being treated differently, even in the healthcare setting.

"Ive had numerous discussions with patients about this. They dont even want to call an ambulance because they fear being judged. They're talking to a nurse, to a tech, or provider and they're called by that dead name. When we call them the dead name their old name that person has to come out all over again, Dr. Ranchero said. They have to experience that shame again.

When treating someone whose experienced discrimination, you can gauge the severity almost as you would with physical burns.

Have they experienced a superficial burn or is it a fourth degree burn and they don't even know the limb is burnt off because theyre so hurt? Dr. Ranchero said, We need to be aware of where that patient is and acknowledge that.

Dr. Ranchero stressed having a climate of dignity and respect, so patients can be themselves.

Set the tone for your clinic, for your practice and your colleagues. What kind of environment are you going to have the first time you meet someone you're unsure of their gender or you don't know their sexual identity?Reassuring the patient theyre in a safe space is a start. But making a connection with the patient will help establish trust. Dr. Ranchero recommends asking how they wish to be addressed and if they feel comfortable sharing their story.

And rather than assuming a transgender pediatric patient isnt capable of making important healthcare decisions, Dr. Ranchero said, We as providers need to ask, Tell me about your journey, about your history. From that, we can gather their insight, their judgment and thought processes.

RN Program Manager of Carle Faith Community Health, Gregory Scott, MS, RN, recognizes how important it is to connect with patients and try to understand their experience with a mindset of whole-person care mind/body/spirit.

We get so focused on the clock Ive got 15 minutes with you thats treating the clock and not the person, Scott said. Take a moment to listen and honestly hear what theyre dealing with.

Dr. Ranchero noted that a common challenge for providers is helping to motivate LGBTQIA+ patients seemingly indifferent to their health. In some cases, this stems from the patients diminished sense of self-worth.

They don't see that their life is worth it and that's where we must begin, Dr. Ranchero said. We say, No, you're a person and youre worth the time and effort. I care about you and your health.

This was something even Dr. Ranchero needed to hear to combat his own self-loathing and feelings of worthlessness.

I stand up for them because someone needed to stand up for me and say, Bernie, you're worthy. You're worthy of medicine, you're worthy of good care and you're worthy of love. And we as providers need to share that compassion for our LGBTQIA patients.

Though even providers who want to be compassionate, fair and respectful can be influenced by unconscious biases.

Our unconscious thought might go against what we know and verbalize. Inside, bias still plays a role, Dr. Ranchero said. But we need to meet patients where they are and not ask them to come to our values and culture.

Scott has noticed how bias can affect patient outcomes and care.

When patients sense our bias, they may never come back, Scott said. We see this escalation of chronic conditions that arent managed well, and patients who dont receive regular annual visits because at some point, they endured a negative experience or trauma.

Giving patients the care they need and treating them with respect is a duty Dr. Ranchero holds sacred.

The most defining moments in my life are when Ive received healing be it physical, psychological, emotional, cognitive, or spiritual. Those are powerful moments. As healers, were called to be conduits, or pathways, for others to receive healing.

Categories: Culture of Quality, Redefining Healthcare, Community

Tags: gender, inclusivity, physicians

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Worthy of medicine, worthy of good care and worthy of love | Carle.org - Carle Health

Josh and Marjorie Harris announce new funding initiative with Penn Medicine – PhillyVoice.com

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Sixers managing partner Joshua Harris and his wife Marjorie have announced a new initiative where they will invest $1 million in companies funded by the Penn Medicine-Wharton Fund for Health. The goal of this undertaking is to improve the livelihood of Philadelphians while focusing on the social determinants of health.

More info from the Sixers is available below:

Philanthropists Josh Harris, managing partner of the Philadelphia 76ers, and his wife, Marjorie Harris, plan to invest $1 million in companies funded by the Penn Medicine Wharton Fund for Health, a collaboration between Penn Medicine and the Wharton Social Impact Initiative of the University of Pennsylvanias Wharton School that invests in early-stage businesses working to improve the social determinants of health of economically disadvantaged Philadelphians. The initiative was first announced in August 2021, with initial plans for a $5 million investment over the following three years.

We are thrilled to continue collaborating with Penn Medicine through the Fund for Health to improve the health of Philadelphia communities, particularly underserved communities that may not have access to the same resources, said Josh Harris, Managing Partner of the Philadelphia 76ers. There are so many factors that contribute to overall health and well-being the goal of this initiative is to invest in innovative companies that are directly addressing the issues that lead to health inequities and support them as they scale.

Social determinants of health include issues such as food insecurity and lack of access to quality healthcare or stable housing, which affect a wide range of health risks and outcomes. Philadelphia County is among the hardest hit cities in the nation by the toll of these disparities, with more than 25 percent of residents living in poverty, 20 percent coping with food insecurity, and nearly 15 percent of its residents uninsured, according to Penn Medicines 2019 Community Health Needs Assessment report.

No ones zip code should determine whether they're able to live a healthy life, but tackling longstanding, systemic problems requires a creative approach and diverse expertise two factors which were at the foundation of our creation of the Fund for Health, said University of Pennsylvania Health System CEO Kevin B. Mahoney. We can make an even greater impact when like-minded collaborators who share this mission also decide, following their own diligence, to make follow-on investments. I am thrilled Josh and Marjorie Harris will be joining forces with us in this way to propel our vision to improve the social determinants of health in our city.

The four companies who this far have received funding are Kinvolved, Lula, Uptrust and RecoveryLink:

Kinvolved, a leader in developing communications software to reduce absenteeism inunderserved school districts, which has subsequently been acquired by PowerSchool, acloud-based K-12 educational software provider.

Lula, which provides a platform and support for small, often family-owned conveniencestories pharmacies and other brick-and-mortar stores to deliver to customers via appslike DoorDash, Grubhub, and Uber EATS.

Uptrust, a customer relationship management tool that helps keep people out of thecriminal justice system by avoiding unnecessary technical violations, like missing courtdates or probation appointments.

RecoveryLink, a telehealth and electronic recovery records platform that improves theavailability and delivery of recovery support services to people experiencing substanceuse and mental health disorders.

This continues a pre-existing relationship between the Harris' philanthropy efforts and and Penn Medicine that previously helped deploy COVID-19 antibody testing to Philadelphia doctors and nurses early in the pandemic.

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Hackensack Meridian School of Medicine Professor and Director of Cardiac Research at Hackensack University Medical Center Named American Heart…

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Taya Glotzer, M.D., FACC, FHRS, honored at the New Jersey Heart Ball on June 10

Taya Glotzer, M.D., FACC, FHRS, has been named the American Heart Associations (AHA) 2022 Researcher of the Year. The award is given to a researcher who is performing cutting-edge research in the field of heart disease and/or stroke.

Research is a cornerstone of the American Heart Association and acknowledging individuals who are making advancements is very important to us, said Peter Cary, executive director for the New Jersey region with the AHA. Dr. Glotzer and her work deserve recognition, and we are so thankful for her dedication to help people live longer, healthier lives.

Dr. Glotzer, who is a professor of medicine at the Hackensack Meridian School of Medicine and the director of Cardiac Research at Hackensack University Medical Center, received the award at the American Heart Association's annual gala, the New Jersey Heart Ball, on June 10.

Dr. Glotzer is one of our supremely talented researcher-physicians, said Robert C. Garrett, CEO of Hackensack Meridian Health. We applaud her achievement.

We are so proud of Dr. Glotzers contributions to the great science and innovation driven by our network, said Ihor Sawczuk, M.D., FACS, president of Academics, Research and Innovation for Hackensack Meridian Health. Our program to promote clinicians engaging in research continues to pay dividends for our patients - and national organizations are noticing.

Widely published and invited regularly by organizations including the American College of Cardiology, American Heart Association, Heart Rhythm Society, and the European Society of Cardiology to present nationally and internationally, Dr. Glotzer has had a longstanding interest in the prevention of stroke, and in investigating the relationship between atrial fibrillation (AF) and stroke.

She published one of the first papers identifying the association of cardiac implanted device detected AF with poor clinical outcomes; stroke and death, in 2003. Dr. Glotzer was the primary investigator of a landmark trial (TRENDS), which showed that a device detected AF longer than 5.5 hours in the recent 30 days doubles the risk of stroke. She was part of the writing group for the Heart Rhythm Society Expert Consensus Statement on Remote Monitoring, and for the European Heart Rhythm Association consensus document on Device Detected Subclinical AF.

In response to the pandemic, Hackensack University Medical Center launched a COVID-specific cardiology research group, of which Dr. Glotzer is a member. The group is collaborating to shed light on COVIDs effect on the heart and blood vessels. In this capacity, Dr. Glotzer found abnormalities on admission EKGs that predicted poor outcomes in patients hospitalized with COVID-19. She presented her findings at the Heart Rhythm Societys 2021 Annual Scientific Sessions.

Dr. Glotzer received her medical degree from the New York University School of Medicine in 1987, and did her residency, cardiology fellowship, and electrophysiology fellowship at NYU Langone Medical Center. She is board certified in clinical cardiac electrophysiology. She has been director of Cardiac Research at Hackensack University Medical Center since 2005, in addition to the full-time practice of cardiac electrophysiology at Hackensack University Medical Center. She is a fellow of the American College of Cardiology, and a fellow of the Heart Rhythm Society. I love seeing and treating my patients, some of whom Ive known for 20 years, Dr. Glotzer said. I feel privileged to be able to take care of them.

For information about Hackensack Meridian Healths cardiovascular services, visit http://www.hackensackmeridianhealth.org/en/Services/Heart-Care. For information about the Hackensack Meridian School of Medicine, visit http://www.hmsom.org/.

ABOUT HACKENSACK MERIDIAN SCHOOL OF MEDICINE

The Hackensack Meridian School of Medicine, the first private medical school in New Jersey in more than 50 years, welcomed its first class of students in 2018 to its On3 campus in Nutley and Clifton. Hackensack MeridianHealthassumed its independent operation in July 2020. The schools vision is that each person in New Jersey, and in the United States, regardless of race or socioeconomic status, will enjoy the highest levels of wellness in an economically and behaviorally sustainable fashion. The Schools unique curriculum focuses on linking the basic science with clinical relevance, through an integrated curriculum in a team-oriented, collaborative environment. The school prides itself on outreach, through programs like the Human Dimension, which is active in communities across New Jersey.

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Hackensack Meridian School of Medicine Professor and Director of Cardiac Research at Hackensack University Medical Center Named American Heart...

MUELLER SPORTS MEDICINE LAUNCHES THE ‘SKY STABILIZER’, A REVOLUTIONARY APPROACH TO PROTECTING HEALTHY ANKLES – Yahoo Finance

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PRAIRIE DU SAC, Wis., June 28, 2022 /PRNewswire/ -- Mueller Sports Medicine, a trusted leader in sports medicine for more than 60 years,has introduced the 'SKY ANKLE STABILIZER'.

The SKY Stabilizer is uniquely engineered to aid in promoting a more stable positioning range, thereby reducing potential for serious injury that could lead to chronic ankle instability.

The SKY Stabilizer, represents remarkably radical out-of-the-box vision and engineering aimed at providing featherlight and streamlined support to athletes with healthy ankles and want to keep them that way.

Ankle sprains are the #1 sport injury and often it only takes a couple of sprains to weaken an ankle joint such that CAI (chronic ankle instability) becomes a significant risk.

Highly specific and refined, the SKY Stabilizer was born from Mueller's focus upon the biomechanics and causal factors of inversion sprains among athletes focused upon jumping vertically, such as basketball and volleyball players. Research revealed an unrecognized opportunity to enhance ankle protection by lessening the tendency for these athletes to let their feet drop while airborne. This in turn helps keep the ankle in a more stable landing posture and less susceptible to dangerous hyper-ranging in scenarios when they land awkwardly, such as on another player's foot.

The SKY Stabilizer is uniquely engineered to aid in promoting a more stable positioning range, thereby reducing potential for serious injury that could lead to chronic ankle instability.The Sky Ankle Stabilizer represents Mueller's disruptive forward thinking,"said John Cayer, President of Mueller Sports Medicine. "It's a radical departure from the traditional approaches to ankle protection.We want athletes to focus on the game without worry of ankle injury."

Mueller's approach is unique because the SKY, 'feels almost invisible', while wearing it. Many athletes tend to forget they have it on.

ABOUT MUELLER SPORTS MEDICINE

Mueller Sports Medicine, Inc. was founded more than 60 years ago by former University of Wisconsin basketball player turned registered pharmacist, Curt Mueller, who coined the term "sports medicine." The company was based on developing better products to protect athletes from injury and enhance their performance and has expanded into the overall health and wellness segment for all audiences.

Story continues

Mueller, which continues to be a family-owned company, was the first company to offer knee braces with the patented Triaxial Hinge (U.S. Patent Nos. 4,726,362 and 4,573,455) designed to properly track the knee joint and provide near-normal motion. Other products include HydraCinn fabric, a moisture-management system that is soft, comfortable, durable and breathable for long term use, Mueller Green, an earth-friendly line of braces and supports, and Mueller TYPHOON Kinesiology Tape, featuring a revolutionary wave pattern adhesive that moves with the skin and muscles. Other brands include Sport Care, Thor, Omniforce, PFTape, Hot Stuff, Hg80 featuring HydraCinn fabric , Stickum, MTape, ProStrips, Athletic Care, Recoil and Quench Gum.

The extensive line of sports medicine products can be found in more than 100 countries.www.muellersportsmed.com.

Cision

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SOURCE Mueller Sports Medicine

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MUELLER SPORTS MEDICINE LAUNCHES THE 'SKY STABILIZER', A REVOLUTIONARY APPROACH TO PROTECTING HEALTHY ANKLES - Yahoo Finance

From BP to sugar, medicines get expensive by up to 30% in this state | Mint – Mint

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As the Indian rupee has continued to plunge against the dollar amid weakness in domestic shares, this has led to a sharp rise in the cost of medicine in the country. According to a report by Live Hindustan, Livemint's sister publication, the prices of medicines have increased by 30% in Uttarakhand. Most of the new batches of medicines available at medical stores are coming with inflated prices in the hilly state, the daily noted.

Pramod Kalani, Head of the Drug Manufacturing Association, said that the raw material of medicines is becoming increasingly expensive in the international market. Due to the strengthening of the dollar against the rupee, the prices of raw materials are also increasing, he said.

The raw material of Paracetamol, which was earlier 5,000 per kg, has now reached 9,000 per kg. Because of this, companies have had to increase the prices of some medicines.

Manish Nanda, Head of the Wholesale Chemist Association, said that in the last month, the price of medicines has increased by 10 to 30%.

State Drug Controller Tajbar Jaggi admitted that drug makers are increasing the prices of medicines because of the hike in crude oil prices. However, he said that the prices of the scheduled medicines which come under price control cannot be increased.

Increase in drug prices

Medicine old rates new rates

Sugar Injection (Ryzodeg) 1024 1126

Sugar Injection (Lentus) 722 794

Sugar medicine (Glycomet) 155 170

Liver Medicine (Udiliv) 580 694

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From BP to sugar, medicines get expensive by up to 30% in this state | Mint - Mint

Dr. Mary Klotman Reappointed Dean of the School of Medicine – Duke Today

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Dear Colleagues,

We are pleased to share with you that Mary E. Klotman, MD, has been reappointed for a second five-year term as dean of the Duke University School of Medicine, vice chancellor for health affairs at Duke University, and chief academic officer for Duke University Health System. Her new term begins on July 1, 2022.

Dr. Klotman has led Duke University School of Medicine through one of the most challenging eras in our more than 90-year history. Thanks to her leadership and to the extraordinary contributions of our faculty and staff, it will also be remembered for its remarkable achievements. Dr. Klotman was first appointed dean of the Duke University School of Medicine in 2017, after serving for nearly seven years with distinction as the chair of Dukes Department of Medicine. Over the past five years, under the leadership of Dean Klotman and in partnership with chairs, center and institute directors, faculty, providers, students, and staff, the School of Medicine has made significant advances in fulfilling the schools priority missions of excellence in patient care, discovery and its translation, education, and community health improvement.

From the outset, Dean Klotman has embraced a One Duke philosophy and commitment to support and service for faculty, staff and students to ensure their success and the continued prominence of the School of Medicine. Currently ranked sixth among all medical schools in the nation for research and third for NIH funding, the school is home to an outstanding community of faculty, staff, students who are recognized nationally as innovators and leaders, and honored for their achievements.

Dean Klotman led the School of Medicine during the COVID-19 pandemic as faculty, staff and students pivoted to focus on urgent needs, making transformative contributions in treatment, testing and surveillance, vaccine development, mitigation and safety guidance, curriculum and training, and community partnerships.

In 2020, under Dr. Klotmans leadership and in partnership with hundreds offaculty, staff, and students, the school began development of its first strategic plan to dismantle racism and advance equity, diversity and inclusion as part of Moments to Movement. Officially launched in 2021, the plan reflects the schools commitment to tangible and sustainable change institutionally and in our broader communities.

In 2021, the School of Medicine, in partnership with Duke University Health System and the PDC, launched a clinical enterprise strategic plan. In spring 2022, a key milestone of that plan was achieved with a formal agreement to create a new integrated clinical practice - to ensure Duke Healths continued leadership in clinical excellence and support of the academic mission.

Dean Klotmans commitment to scientific excellence is reflected in efforts to retain and recruit outstanding research faculty and provide ongoing support to the research community. During her tenure, eight new faculty scholars have been recruited as part of the Duke Science and Technology initiative, and, since 2017, the School of Medicine has constructed new state-of-the-art research facilities including the Chesterfield Building, the Medical Science Research Building (MSRB) III, and Dukes first research campus in Research Triangle Park: Duke Research & Discovery@RTP.

A national leader in science and academic medicine, Dean Klotman is a member of the National Academy of Medicine and a fellow in the American Academy of Arts and Sciences. She is past president of the Association of American Physicians and past president of the Association of Professors of Medicine. A widely-acknowledged authority on HIV, Dean Klotman and her lab are focused specifically on HIV-associated kidney disease. Most recently, she and her team have been defining the role of integrase-defective lentiviral vectors for the delivery of an HIV vaccine.

We could not be more pleased to have Dr. Klotman at the helm of the School of Medicine. Please join us in congratulating her on this well-deserved reappointment.

Sincerely,

A. Eugene Washington, MDChancellor for Health Affairs, Duke UniversityPresident and CEO, Duke University Health System

Sally Kornbluth, PhDProvost, Duke University

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Dr. Mary Klotman Reappointed Dean of the School of Medicine - Duke Today

Nuclear Medicine Market Size, Scope and Forecast | General Electric Co., Bayer AG, Lantheus Medical Imaging Medtronic and Cardinal Health Inc. …

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New Jersey, United States This Nuclear Medicine Market research examines the state and future prospects of the Nuclear Medicine market from the perspectives of competitors, regions, products, and end Applications/industries. The Worldwide Nuclear Medicine market is segmented by product and Application/end industries in this analysis, which also analyses the different players in the global and key regions.

The analysis for the Nuclear Medicine market is included in this report in its entirety. The in-depth secondary research, primary interviews, and internal expert reviews went into the Nuclear Medicine reports market estimates. These market estimates were taken into account by researching the effects of different social, political, and economic aspects, as well as the present market dynamics, on the growth of the Nuclear Medicine market.

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Key Players Mentioned in the Nuclear Medicine Market Research Report:

General Electric Co., Bayer AG, Lantheus Medical Imaging Medtronic and Cardinal Health Inc.

The Porters Five Forces analysis, which explains the five forces: customers bargaining power, distributors bargaining power, the threat of substitute products, and degree of competition in the Nuclear Medicine Market, is included in the report along with the market overview, which includes the market dynamics. It describes the different players who make up the market ecosystem, including system integrators, middlemen, and end-users. The competitive environment of the Nuclear Medicine marketis another major topic of the report. For enhanced decision-making, the research also provides in-depth details regarding the COVID-19 scenario and its influence on the market.

Nuclear MedicineMarket Segmentation:

Nuclear Medicine Market, By Type

Diagnostic Therapeutic

Nuclear Medicine Market, By Modality

SPECT PET Alpha-emitters Beta-emitters Brachytherapy

Nuclear Medicine Market, By Application

Oncology Cardiology Neurology Thyroid

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Nuclear Medicine Market Report Scope

Key questions answered in the report:

1. Which are the five top players of the Nuclear Medicine market?

2. How will the Nuclear Medicine market change in the next five years?

3. Which product and application will take a lions share of the Nuclear Medicine market?

4. What are the drivers and restraints of the Nuclear Medicine market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Nuclear Medicine market throughout the forecast period?

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Nuclear Medicine Market Size, Scope and Forecast | General Electric Co., Bayer AG, Lantheus Medical Imaging Medtronic and Cardinal Health Inc. ...