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Genes may influence our successes and failures in life, according to Professor Kathryn Paige Harden – ABC News

Posted: July 19, 2022 at 2:32 am

Many of us like to think if we work hard, we'll succeed. But what if there's something we have no control over that could influence how successful we are?

Recent research suggests our genes can influence how far we go in school and how much money we make as adults.

Kathryn Paige Harden, a professor of psychology and behaviour geneticist from the University of Texas, says acknowledging "genetic luck" could be used to help create a more equitable society.

However, her book on the subject, The Genetic Lottery: Why DNA Matters for Social Equality, has caused considerable debate, with some even calling it "dangerous".

Professor Harden understands the criticism. She says the history of the genetic sciences and the "atrocious eugenic views" held by many of the field's forefathers make it a difficult field to navigate.

"There has never been a time, from Darwin on, in which the discussion of genetics or evolution or heredity in relation to humans has not been something that causes anxiety and controversy," she says.

However, she arguesit's time to "reclaim" the field and embrace the idea that, although they may not fully determine our destiny, our genes do matter.

Until the commencement of the international scientific research project, theHuman Genome Project (HGP), in 1990, there was little understanding of what humans looked like on a molecular level. This meant looking at our genetic similarities and differences was next to impossible.

By 2003, when the HGP wrapped up, researchers had successfully mapped over 90 per cent of the human genome. And in March 2022, the final pieces of the human genome puzzle were put in place.

Theoretically, there is now a set of instructions forhow to build a human being.

"Every human has in their cells 23 pairs of chromosomes, unless you have a condition like [Down syndrome], in which you've inherited an extra chromosome," Professor Harden says.

"All these chromosomes, your DNA, are made up of four DNA letters:G, C, T and A.

"Humans are more than 99 per cent genetically similar," Professor Harden says, adding that "most of what human DNA does is make a human body".

It's the remaining portion less than one per cent that differs between people that scientists like Professor Harden are interested in.

She says most studies focus on the single DNA letter differences between people known assingle-nucleotide polymorphisms orSNPs, which are the most common type of genetic variation found among people.

"I might have a T in a certain spot, and you might have a C in a certain spot," she says.

"[There can be] millions of [these genetic variants] scattered throughout your entire genome."

Around two decades ago, scientists began to look at which SNPswere associated with specific outcomes.

"[For instance] if the outcome we are interested in is height, we might say which genetic variants (SNPs) are more common in tall people versus short people," Professor Harden says.

Initially these studies focused on things like high cholesterol, macular degeneration or type 2 diabetes.

This research has helped identify genetic variants associated with an increased susceptibility to developing these conditions later in life.

But since then, the focus of these studies has shifted.

Researchers are now looking at more socially focused outcomes, such ashow far someone went in school, how much money they make and if they've ever been addicted to opiate drugs.

"A lot of people were sceptical that [this research] would work," Professor Harden says.

However, studiesshow patterns of genetic correlations that are related to these psychological behavioural outcomes.

When it comes to how well kids and adolescents do in school, Professor Harden says we already know all things aren't equal.

"We have a ton of research about that from educational and developmental psychology," she says.

We know that poverty and disadvantage outside of school impact students' educational outcomes.

But Professor Harden argues that cognitive ability is another part of the equation.

"If you have better working memory, better visual spatial reasoning [or]a stronger vocabulary, school is easier for you," she says.

Non-cognitive factors also come into it. One of those is personality, something thatProfessor Harden is veryinterested in.

"There are personality traits that might make school easier or harder," she says.

Things like impulsivity, how organised you are and how persistent you are. And these traits are at least partly shaped by our genes, she says.

The relationship between genetics and educational and economic success is complex. Professor Harden says people often try and simplify it by comparing it to a poker game.

"There's the genes or the hand you get dealt, but there's still how you play that hand," she says.

Butthe effect of genes on things like personality means this metaphor can break down.

"Our genes are also influencing how we play the hand we're dealt. It influences how motivated we are, how [much we plan], how much impulse control we have," she says.

"It makes this line between what's effort and agency and what's [genetic] luckkind of impossible to tease apart."

Professor Harden says there's a problematic lack of diversity in the research so far on this topic.

"Right now, the vast majority of information we have about the human genome comes from one narrow slice of the global population and that's people with Northern European ancestry," she says.

"The most common study is of people who self-identify as white British."

She argues that this isn't only "inequitable" but it "hurts the science".

"We're neglecting an enormous pool of genetic diversity and variety," Professor Harden says.

She believesexpanding the diversity of genetics is a great opportunity for future work in the field. But in the meantime, we're left with studies that don't necessarily apply to everyone.

Given genes are immutable, Professor Harden says a lot of people have asked why the recent studies matter so much.

She says this isbecause there is scope to intervene and make a difference.

"Just because something is genetic doesn't mean we can't intervene on it environmentally."

One example she highlights is how family therapyis used to help treat alcohol abuse problems in adolescents.

"[Genetically speaking], not every teenager is equally likely to develop an alcohol abuse problem. Some of that genetics has to do with how your body metabolises alcohol, but some of it has to do with personality," she says.

"Do you tend to like loud, rowdy friends? Do you like to go to parties wheresubstances will be on offer?"

Professor Harden says randomised controlled trials have shown that family therapy, which aims to improve parent-teenagerrelationships and communication, isan effective treatmentandhelps kids who are "most genetically at risk".

"That's because one of the pathways between their genetic risk and their addiction is through their social environment."

The possibility of making a difference is posed as a question in Professor Harden's book.

"How can public spaces, working conditions, access to medical care and legal codes and social norms be reimagined such that the arbitrariness of nature is not crystallised into an inflexible caste system?" she writes.

And some people are looking for the answers.

Professor Harden says although there's been a bit of "pushback" from fellow academics, there's also beena lot of interest from policymakers and governmental institutions

"[They] have reached out to me to say: 'We want to hear more about this'. I think a lot of people are hungry for new tools and new solutions."

This conversation between Rob Brook and Kathryn Paige Harden was originally recorded as part of UNSW Centre for Ideas and broadcast on ABC RN's Big Ideas.

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People’s University Is All About Dinosaurs This Summer – Wheeling Intelligencer

Posted: July 19, 2022 at 2:32 am

WHEELING Its the summer of dinosaurs, and the Ohio County Public Library in Wheeling invites patrons to learn all about the amazing prehistoric creatures in an eight-week series.

The new dinosaur series will feature paleontolgists and students of paleontology from Pittsburghs venerable Carnegie Museum of Natural History, discussing topics ranging from defining what dinosaurs actually were, to how they are related to modern birds and reptiles, to how and why they became extinct. The series will conclude with a behind-the-scenes field trip guided by the museums principal dinosaur researcher himself, Dr. Matthew Lamanna.

For inquiries and to register for the series, call the library at 304-232-0244, visit http://www.ohiocountylibrary.org, send an email, or visit the librarys reference desk.

All classes will take place on Thursday evenings. The full class schedule for Peoples University Dinosaurs at the Ohio County Public Library will be as follows:

Class 1: July 21 at 7 p.m. What is a Dinosaur?

A fun, interactive introduction into what is and isnt a dinosaur. Many people exclude things like birds from their definition of a dinosaur, but include things like crocodiles, turtles, pterosaurs, and sometimes even mammoths. This lecture would clarify misunderstandings of what makes something a dinosaur, like the fact that something doesnt have to be extinct to be a dinosaur but they do need their legs to be positioned beneath their bodies.

Class 2: July 28 at 7 p.m.

The Dinosaur Family Tree

With the definition of what dinosaurs are already established, we will explore the evolutionary history of dinosaurs, including the many different groups of dinosaurs and how they are related. Everything from ornithomimids to hadrosaurs are fair game!

Class 3: Aug. 4 at 7 p.m. Tectonics & Dinosaur Dispersal

Discover how the position of the continents changed over prehistory and how that impacts where dinosaurs are discovered today. There are species of dinosaur that are found on multiple continents, demonstrating how much closer the continents were at the time.

Class 4: Aug. 11 at 7 p.m. Dinosaur C.S.I.

Dinosaurs left more behind than just their bones and skin. They also left footprints, coprolites and other evidence of their day-to-day life.

We will examine different types of dinosaur fossils and how each informs paleontologists about dinosaur behavior, just like how crime scene investigators use physical evidence to piece together what happened.

Class 5: Aug. 18 at 7 p.m. Dinosaur Species of Jurassic Park

What do Velociraptor, Brachiosaurus, Triceratops, Dilophosaurus, and, of course, Tyrannosaurus rex have in common? They all became movie stars in the internationally popular film, Jurassic Park. Even more species like Allosaurus and Stegosaurus appeared in the movies sequels. Giganotosaurus appears in the latest installment, Jurassic World: Dominion, released this summer. But were their portrayals realistic according to the latest science? We will explore this question.

Class 6: Aug. 25 at 7 p.m. The Evolution of Flight

We will take to the air to discover how feathered dinosaurs became the progenitors of birds and unravel the avian link to dinosaur species such as Archaeopteryx and Microraptor. Well also take a look at pterosaurs.

Class 7: Sept. 1 at 7 p.m. The End of Dinosaurs and Rise of Mammals

Mammals originated at the same time as dinosaurs but remained overshadowed until the non-avian dinosaurs went extinct. What led to mammals subsequent success? Trace the rise of mammals from humble origins to charismatic megafauna, and discover some of the unique traits that have helped them thrive in changing habitats on land and at sea.

Class 8: Sept. 8 at 6 p.m. Finale Field Trip to Carnegie Museum of Natural History

Participants who attend all of the first seven classes will get preference for the field trip, as we are limited to 20 people. If more than 20 qualify, we will draw names.

Attendees will get a behind the scenes look at the Dinosaurs in Their Time exhibition at the Carnegie Museum of Natural History in Pittsburgh. Those interested will be responsible for their own transportation to and from the museum, where we will meet at 6 p.m. for the tour. It will last about 1 hour. This exhibition is home to dozens of real, original fossils displayed in scientifically accurate reconstructions of their ancient habitats.

ABOUT THE INSTRUCTORS

Lindsay Kastroll will be the instructor for Classes 1-4. She is a paleontology student and museum volunteer with a special interest in dinosaurs. Following her recent graduation from California University of Pennsylvania with degrees in biology and geology, she will be attending a masters program in Biological Sciences at the University of Alberta starting in Fall 2022 where she will complete research on ornithischian dinosaurs: think things like Triceratops, Ankylosaurus, and Stegosaurus. She got her start volunteering with the Carnegie Museum of Natural History writing Mesozoic Monthly, a series of deep dives on prehistoric creatures for the museum blog.

Taylor McCoy will instruct Classes 5-6. He is a vertebrate paleontology volunteer at the Carnegie Museum of Natural History under Dr. Matt Lamanna. His experience there includes community outreach through science communication and fossil restoration. McCoy also has field experience working with Dr. Thomas Carr in Montana, excavating and prospecting fossils from the late Cretaceous.

Dr. A. R. West will instruct Class 7. West holds a PhD in paleontology from Columbia University and a BA in organismal biology from the Univ. of Cambridge, UK. Dr. West moved to Pittsburgh to complete a postdoctoral fellowship at Carnegie Museum of Natural History in the Section of Paleontology and the Section of Mammals. They now work in the department of Biological Sciences at the University of Pittsburgh, where they teach classes on molecular genetics, evolution and science communication. West has carried out paleontology fieldwork in several different states, the UK and Antarctica.

Dr. Matthew Lamanna will serve as instructor and museum tour guide for Class 8. He is the Mary R. Dawson Associate Curator of Vertebrate Paleontology and the principal dinosaur researcher at Carnegie Museum of Natural History in Pittsburgh. He received his bachelor of science degree from Hobart College and his master of science degree and Ph.D. from the University of Pennsylvania. He has directed or co-directed field expeditions to Antarctica, Argentina, Australia, China, Croatia, Egypt and Greenland that have resulted in the discovery of numerous new species of dinosaurs and other animals from the Cretaceous Period. Lamanna served as chief scientific advisor to Carnegie Museums $36 million Dinosaurs in Their Time exhibition and has appeared on television programs for PBS (NOVA), Discovery Channel, History Channel, A&E, the Science Channel and more.

For inquiries about this new Peoples University series and to register, call the library at 304-232-0244, email the library staff or visit http://www.ohiocountylibrary.org.

The Peoples University is a free series open to the public. Guests are welcome to attend as many classes as they wish. There are no tests or other requirements.

The first 50 attendees to register and attend the first class will get a free official Peoples University Dinosaurs T-shirt, which can be found at Zazzle.com. Those people will also receive free dinosaur reference books recommended by our experts, including The Rise and Fall of the Dinosaurs: A New History of a Lost World and Dinopedia, an illustrated, pocket-friendly encyclopedia of all things dinosaurian.

Both books are complimentary for attendees, who will also receive a dinosaur tote bag.

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Lecturer in Biology (Education Focused) job with UNIVERSITY OF SYDNEY | 300815 – Times Higher Education

Posted: July 19, 2022 at 2:32 am

About the opportunity

The School of Life and Environmental Sciences (SOLES) is seeking to appoint a Lecturer in Biology (Education Focused) to be based at the Camperdown Campus. This is a wonderful opportunity to join the University of Sydney Australias first university at an exciting and innovative time with the development of a new strategic plan, which will build upon our outstanding global reputation of education and research excellence. SOLES brings together academics from across a broad range of disciplines to understand and solve important global challenges. Key majors include biochemistry, ecology, genetics, marine biology, microbiology, molecular biology, plant science, soil science, animal science and agriculture. This exciting new education-focused role will work closely with the First year Biology teaching team in contributing to the development and delivery of our three first year biology units.Preference may be given to applicants with experience in teaching cell, molecular and human biology. The appointee will deliver content in first year biology and developstrategies thatfoster active engagementof students to ensure excellence in teaching and learning outcomes.

The successful applicant will have a demonstrated track record in educational excellence in the development and delivery of interactive content in first year Biology and Life Science units that enriches the existing strengths and expertise within the school. The successful applicant will also have a track record in implementation of scholarly and evidence-based teaching innovations to create a safe and dynamic learning environment for students.Their track record will include evidence of success in improving the student experience and embedding of digital and other technological innovations in Biology (Life Science) teaching and learning and working effectively in an academic team.We are intent on implementing scholarly approaches to create a world-class curriculum for incoming students transitioning from school to university to enable them to thrive in their studies.

About you

The University values courage and creativity; openness and engagement; inclusion and diversity; and respect and integrity. As such, we see the importance of recruiting talent aligned to these values and are looking for anLecturer in Biology (Education Focused) who has:

To keep our community safe, please be aware of our COVID safety precautions which form our conditions of entry for all staff, students and visitors coming to campus.

Sponsorship / work rights forAustralia

Please note: Visa sponsorship is not available for this position. For a continuing position, you must be an Australian or New Zealand citizen or an Australian Permanent Resident.

Pre-employment checks

Your employment is conditional upon the completion of all role required pre-employment or background checks in terms satisfactory to the University. Similarly, your ongoing employment is conditional upon the satisfactory maintenance of all relevant clearances and background check requirements. If you do not meet these conditions, the University may take any necessary step, including the termination of your employment.

EEO statement

At the University of Sydney, our shared values include diversity and inclusion and we strive to be a place where everyone can thrive. We are committed to creating a University community which reflects the wider community that we serve. We deliver on this commitment through our people and culture programs, as well as key strategies to increase participation and support the careers of Aboriginal and Torres Strait Islander People, women, people living with a disability, people from culturally and linguistically diverse backgrounds, and those who identify as LGBTIQ. We welcome applications from candidates from all backgrounds.

How to apply

Applications (including a cover letter, CV, and any additional supporting documentation) can be submitted via the Apply button at the top of the page.

If you are a current employee of the University or a contingent worker with access to Workday, please login into your Workday account and navigate to the Career icon on your Dashboard. Click on USYD Find Jobs and apply.

For a confidential discussion about the role, or if you require reasonable adjustment or support filling out this application, please contact Simon Drew, Recruitment Operations, Human Resources at recruitment.sea@sydney.edu.au

The University of Sydney

The University reserves the right not to proceed with any appointment.

Applications Close

Sunday 07 August 2022 11:59 PM

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Radiologists hope to use AI to improve readings – University of Miami: News@theU

Posted: July 19, 2022 at 2:32 am

The Miller School of Medicine Department of Radiology is working with the Universitys Institute for Data Science and Computing to design an artificial intelligence tool that could help them diagnose patients in a more individualized way.

Over the years, new technology has helped radiologists diagnose illnesses on a multitude of medical images, but it has also changed their jobs.

While in the past these physicians spent more time speaking with patients, today they spend most of the time in the reading rooma dark space where they scrutinize images alongside a patients electronic medical records and other data sourcesto diagnose an illness.

A radiologists job is often solitary. And it is a trend that University of Miami Miller School of Medicine radiologists Dr. Alex McKinney and Dr. Fernando Collado-Mesa hope to change.

The two physicians have been working with the Universitys Institute for Data Science and Computing (IDSC) to create an artificial intelligence toolbox that will draw on a massive database of deidentified data and medical images to help doctors diagnose and treat diseases based not only on imaging data but by also considering a patients unique background and circumstances. This would include risk factors, like race and ethnicity, socioeconomic and educational status, and exposure. The physicians say it is a necessary innovation at a time when narrow artificial intelligence in radiology is only able to make a binary decision such as positive or negative for one disease, rather than scanning for a host of disorders.

We believe the next iteration of artificial intelligence should be contextual in nature, which will take in all of a patients risk factors, lab data, past medical data, and will help us follow the patient, said McKinney, who is also the chair of the Department of Radiology. It will become a form of augmented interpretation to help us take care of the patient.

According to Collado-Mesa, this toolbox will not just say yes or no, disease or no disease. It will point to the data around it to consider a variety of issues for each individual patient, to put its findings into a context, including future risk.

Current artificial intelligence tools are also limited to a specific type of medical image, and cannot, for example, analyze both MRI (magnetic resonance imaging) and ultrasound at the same time. In addition, the patient data that is used in these diagnosis tools is typically not inclusive of a range of demographic groups, which can lead to a bias in care. Having a tool that draws upon the examples of millions of South Florida patients, while maintaining their privacy, will help radiologists be more efficient and comprehensive, McKinney noted.

Right now, there is just so much data for radiologists to sift through. So, this could help us as our tech-based partner, McKinney added.

All of these factors led Collado-Mesa and McKinney to try and create a better alternative, and they spoke with IDSC director Nick Tsinoremas, also a professor of biochemistry and molecular biology. Tsinoremas and IDSCs advanced computing team came up with the idea of utilizing an existing toolcalled URIDEa web-based platform that aggregates deidentified patient information for faculty researchand adding in the deidentified images from the Department of Radiology.

They hope to unveil a first version of the toolbox this summer and plan to add new elements as more imaging data is added. It will include millions of CT scans, mammograms, and ultrasound and MRI images, along with radiographs, McKinney pointed out.

We dont want to rush this because we want it to be a high-quality, robust toolbox, said Collado-Mesa, an associate professor of radiology and breast imaging, as well as chief of innovation and artificial intelligence for the Department of Radiology.

Both physicians and Tsinoremas hope that the artificial intelligence tool will help answer vital research questions, like: what risk factors lead to certain brain tumors? Or, what are the most effective treatments for breast cancer in certain demographic groups? It will also use machine learning, a technique that constantly trains computer programs how to utilize a growing database, so it can learn the best ways to diagnose certain conditions.

Creating this resource can help with diagnosis and will allow predictive modeling for certain illnesses, so that if a person has certain image characteristics and clinical information that is similar to other patients from this database, doctors could predict the progression of a disease, the efficacy of their medication, and so on, Tsinoremas said.

To ensure the toolbox will be unbiased, the team is also planning to add more images and data of all population groups in the community, as it is available, as well as to monitor the different elements constantly and systematically within the toolbox to make sure it is performing properly.

The radiologists plan to focus first on illnesses that have a high mortality or prevalence in the local population, like breast cancer, lung cancer, and prostate cancer, and to add others with time.

The technology could allow them to spend more time with patients and offer more personalized, precision-based care based on the patients genetics, age, and risk factors, according to both physicians.

Artificial Intelligence has the potential to advocate for the patients, rather than a one-size-fits-all approach to medicine based on screening guidelines, McKinney said. This could help us get away from that, and it would hopefully offer more hope for people with rare diseases.

But as data is added in the future, the researchers hope to expand their work with the tool. And they hope that physicians across the University will use it to conduct medical research, too.

This is a resource that any UM investigator could potentially access, provided that they have the approvals, and it could spark a number of different research inquiries to describe the progression of disease and how patients respond to different treatments in a given time periodthese are just some of the questions we can ask, Tsinoremas said.

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Governors Try to Find Common Ground in a Divided America – Governing

Posted: July 19, 2022 at 2:30 am

(TNS) In a summer of rising tensions among U.S. states deeply divided over the rights of women, voters and gun owners, 19 of the nation's governors have gathered in Maine to find common ground on less divisive issues, such as advancing computer science education in public schools and assisting the post-pandemic recovery of the tourism industry.

Gov. Janet Mills welcomed her counterparts to Portland for the semi-annual meeting of the National Governors Association, a nonpartisan entity representing the chief executives of the fifty-five states, territories, and commonwealths, with a pitch to bridge partisan divisions.

"(Mainers) are people of all political parties, bound by the shared belief that their government should work for them," Mills said. "That belief is in our blood, bred by generations of Maine leaders like Margret Chase Smith, Edmund S. Muskie, Bill Cohen, George Mitchell, and Olympia Snowe, people who believed in putting their country before their political party."

Speaking to reporters after the first plenary, North Dakota Gov. Doug Burgum said the bipartisan forum was valuable. "We're a group that has the shared experience in terms of leading and there are opportunities for bipartisan cooperation," the Republican said. "Some of the national issues affect us differently, but all of us have similar challenges even if we have different dynamics in terms of our legislatures and local politics. But it's great comparing notes."

Outgoing NGA Chair Asa Hutchinson, Republican governor of Arkansas, announced that fifty of the 55 states, territorial and commonwealth governors had signed a compact he has been promoting committing to expand computer science education in public schools. The pledge, endorsed by Mills, commits the governors to implement at least one of thirteen policy initiatives meant to increase the number and demographic diversity of students studying computer science.

New Hampshire-based inventor Dean Kamen, whose engineering powerhouse DEKA is pioneering the rapid production of human organs grown from a patient's own cells, urged governors to boost the supply of young people interested in science and technical fields by contributing to his ongoing effort to make robotics competitions into a sport as popular as scholastic football, basketball or baseball.

Kamen's observation, back in the late 1980s, was that the emerging shortage of science, technology and engineering experts was a cultural problem: schools and their students didn't celebrate STEM, they celebrated sports. "We know a model that works sports. Let's create a sport around science and engineering," he recalled to the governors.

The result was FIRST, a robotics competition for K-12 students that's grown from 23 teams in 1989 to some 50,000 teams fielding more than 700,000 student competitors in 113 countries. Maine alone has 633 teams. Backed by donations from tech giants, the nonprofit has dozens of employees and $70 million in assets.

He wants the governors' help including Mills to further expand the model and increase its profile in an effort to give the sport soccer-like visibility. Students who become passionate about STEM, he reasons, will transform their own prospects and that of the country.

"I'd say, Gov. Mills, you're a tiny state, and I bet you don't have 100 high schools, and if you put a line in your budget that said we're going to have a FIRST team in every high school," Kamen told the Press Herald. "And I want every other governor to say: no, I want to be the first one to claim that victory."

The CEO of computer chip giant Intel, Pat Gelsinger, repeatedly urged the assembled governors to press their states' congressional delegation to pass the core provisions of the CHIPS Act: $52 billion in incentives for firms like Intel to shift semiconductor manufacturing back to the United States. Gelsinger, who has been lobbying for action on the long-stalled bill for months, implied chipmakers would invest in overseas manufacturing instead if Congress didn't act before the August recess.

"Call your senators we need this done before the August recess," Gelsinger said. "I and others will make decisions ... to decide if meaningful portions (of future manufacturing) will be on American soil or not."

While the tourism sector officials including a representative of the U.S. Travel Association encouraged governors to help fund marketing efforts, Mills and two other New England governors Republicans Chris Sununu of New Hampshire and Charlie Baker of Massachusetts noted the enormous problem of tourism workforce housing, which has been exacerbated by the spread of short-term rentals via outlets like VRBO and Airbnb.

"This seems to be a major impediment to securing the workforce for the industry," Mills said.

Baker said his government had started building worker housing on Cape Cod because short-term rentals had "sucked up all the housing" there. Sununu related how, as a resort operator in the Waterville Valley of New Hampshire in 2016, he had been forced to buy another hotel just to house his workers. This prompted Utah Gov. Spencer Cox, a Republican, to observe that the country was absurdly "turning houses into hotels and hotels into housing."

Earlier Thursday the governors held a closed-door session with officials from the Defense Department and Federal Emergency Management Agency to foster more federal-state cooperation in dealing with wildfires and droughts, Cox said in a tweet.

At lunch also closed to the media L.L. Bean CEO Steve Smith spoke to the governors "about the health benefits of outdoor recreation," according to a tweet by the NGA.

The governors convene again Friday, July 15, to appoint new officers and to discuss boosting early childhood literacy (virtually) with Dolly Parton, who created a nonprofit around this issue.

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What to Know About Gene Therapy – Biotech – HIT Consultant

Posted: July 19, 2022 at 2:29 am

Gene therapy is making a world where cancer and AIDS can be cured, and a person can alter their genetic makeup, changing the direction of their own and their offsprings evolution in the process. However, the effects of gene therapy are long-lasting, and may impact both your and the health of your future children.

What Is Gene Therapy?

Utilizing a gene or genes to treat or cure a disease or medical condition is known as gene therapy. Gene therapy frequently involves replacing a damaged gene in a patients cells with a healthy copy or inserting new copies of a damaged gene.

Instead of drugs or surgery, gene therapy procedures allow doctors to treat a problem by changing a persons genetic composition. The first gene therapy technique, also known as gene transfer or gene addition, was created to:

Add a new gene to cells to aid in treating a disease

Introduce a healthy gene copy to replace the disease-causing altered copy

Later research helped to advance gene therapy methods. A more recent method, genome editing, takes a different tack in addressing genetic discrepancies. Genome editing uses molecular tools to alter the DNA already present in cells rather than introducing new genetic material. Research on genome editing aims to:

Restore a genes normal function

Correct the genetic change that underlies an illness

Activate a gene that isnt working correctly

Eliminate a section of DNA that interferes with gene activity and causes illness

However, gene therapy is still a field that primarily exists in research labs, and its application is still being tested. The majority of trials take place in Europe, US, and Australia. The treatment is extensive and is being tested to be used to treat acquired genetic diseases like cancer and some viral infections like AIDS, as well as diseases brought on by recessive gene defects, including cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia.

Types of Gene Therapy

Gene therapy comes in various forms, including:

Gene editing: Gene editing aims to remove undesirable genes or fix mutated genes

Cellular gene therapy: The patients cells are taken out, genetically altered (typically via a viral vector), and then put back in

Plasmid DNA: It is possible to genetically modify circular DNA molecules to deliver healing genes into human cells

Viral vectors: Certain gene therapy items are made from viruses because they naturally possess the potential to introduce genetic material into cells. These altered viruses can be employed as vectors to transport therapeutic genes into cells once viruses have been altered to reduce their capacity to spread infectious diseases

Ethical Issues in Gene Therapy

The idea of genetically altering genes has long been the subject of contentious debate in the scientific community. When new techniques are developed, bioethics is always present to evaluate the procedures hazards and moral ramifications. Genetic therapy in somatic cells is widely accepted in the scientific community, particularly in severe diseases like cystic fibrosis and Duchenne muscular dystrophy.

For instance, the first experiment for modifying healthy human embryos was allowed in the United Kingdom. On the other hand, American research organizations remained conservative, restating their stance that they do not support this kind of trial and saying that they must wait for advancements in both the methods and the definitions of ethical issues.

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Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and…

Posted: July 19, 2022 at 2:29 am

New York, July 15, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions : Industry Trends and Global Forecasts, 2022-2035" - https://www.reportlinker.com/p06292885/?utm_source=GNW To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors). Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain. In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods. For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics. Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation. Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

SCOPE OF THE REPORTThe Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

Amongst other elements, the report features:A detailed overview of the overall market landscape of gene therapies, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene / molecule targeted, type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).A detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.An elaborate discussion on the various commercialization strategies that have been adopted by drug developers engaged in this domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.Detailed profiles of marketed and late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio strength, dosage and manufacturing details, as well as details related to the developer company.A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product, target therapeutic area and deal multiples.An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by companies that are engaged in this domain.An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.An analysis of the startup companies engaged in this domain (established between 2017-2022) based on year of experience.A detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength.An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic areas (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were Influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:Buel Dan Rodgers (Founder and CEO, AAVogen)Sue Washer (President and CEO, AGTC)Patricia Zilliox (President and CEO, Eyevensys)Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))Adam Rogers (CEO, Hemera Biosciences)Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))Al Hawkins (CEO, Milo Biotechnology)Jean-Phillipe Combal (CEO, Vivet Therapeutics)Robert Jan Lamers (former CEO, Arthrogen)Tom Wilton (former CBO, LogicBio Therapeutics)Michael Triplett (former CEO, Myonexus Therapeutics)Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)Cedric Szpirer (Executive and Scientific Director, Delphi Genetics)Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory)Jeffrey Hung (CCO, Vigene Biosciences)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

Th secondary sources of information include:Annual reportsInvestor presentationsSEC filingsIndustry databasesNews releases from company websitesGovernment policy documentsIndustry analysts views

While the focus has been on forecasting the market till 2035, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWEREDWho are the key industry players engaged in the development of gene therapies?How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?Which types of vectors are most commonly used for effective delivery of gene therapies?What are the key regulatory requirements for gene therapy approval, across various geographies?Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?What are the key value drivers of the merger and acquisition activity in the gene therapy industry?Who are the key stakeholders that have actively made investments in the gene therapy domain?Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and ex vivo and in vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the various advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 1150 gene therapies that are currently approved or are in different stages of development. It features a detailed analysis of the therapies, based on several relevant parameters, such as key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene / molecule targeted, type of gene delivery method used (ex vivo and in vivo), type of therapy (gene augmentation, oncolytic viral therapy, immunotherapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and type of therapy.

Chapter 7 provides a detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe, Asia-Pacific, and Middle East and North Africa region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that have been adopted by therapy developers engaged in this domain across key commercialization stages, including prior to drug launch, during drug launch and post-launch stage. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2022.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II / III and above). Each drug profile provides information on the development timeline of the therapy, current developmental status, route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, affiliated technology, patent portfolio strength, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline candidates that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic diseases, metabolic diseases, neurological diseases, oncological diseases and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed / granted). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product, target therapeutic area and deal multiples.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and secondary offerings) received by the companies between 2015 and 2022, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents an analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 presents a detailed analysis of the start-up companies engaged in the field of gene therapy, established between 2017-2022, based on year of experience.

Chapter 19 provides a detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. In addition, it also a detailed analysis of the big pharma players based on several parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used.

Chapter 20 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 21 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2035. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II / III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic area (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world).

Chapter 22 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 23 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 24 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 25 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Buel Dan Rodgers (Founder and CEO, AAVogen), Sue Washer (President and CEO, AGTC), Patricia Zilliox (President and CEO, Eyevensys), Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics)), Adam Rogers (CEO, Hemera Biosciences), Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela)), Al Hawkins (CEO, Milo Biotechnology), Jean-Phillipe Combal (CEO, Vivet Therapeutics), Robert Jan Lamers (former CEO, Arthrogen), Tom Wilton (former CBO, LogicBio Therapeutics), Michael Triplett (former CEO, Myonexus Therapeutics), Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics), Cedric Szpirer (Executive and Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory), and Jeffrey Hung (CCO, Vigene Biosciences). In addition, a brief profile of each company has been provided.

Chapter 26 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 27 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06292885/?utm_source=GNW

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Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and...

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Systemic gene therapy with thymosin 4 alleviates glomerular injury in mice | Scientific Reports – Nature.com

Posted: July 19, 2022 at 2:29 am

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Systemic gene therapy with thymosin 4 alleviates glomerular injury in mice | Scientific Reports - Nature.com

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The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development – Contract Pharma

Posted: July 19, 2022 at 2:29 am

Recently, a patient centricity concept has spread across all corners of the healthcare community with a straightforward goal: to improve and save patients lives through innovative and effective drug therapies adapted to patients' individual needs. However, for this concept to truly come to life, it is necessary to count on temperature-controlled supply chain logisticslike CRYOPDP to turn what could be a complex operation into a highly efficient process. This is extremely vital, because a patients life could literally be on the line with every shipment.Patient centricity in the clinical trial community involves meeting the patients where they are.The clinical trial industry is globalising and demands international healthcare logistics partners that can serve its growing and continuously evolving needs. For decades, temperature-controlled logistics experts have operated behind the scenes with patients barely aware of the hard work that went into their medicines journey. Thanks to the Covid-19 vaccine distributions impressive performance and its universal effect on people's health across the globe, it has become apparent to the world just how critical and important a logistics providers mission is for both patients and the industry.The endless pandemic lockdowns meant that patients could not travel to hospital sites for clinical trials due to travel and access restrictions. This led to a seismic shift where sponsors quickly moved to a direct-to-patient model to try to continue trials and keep patient treatments on schedule. As part of our response to this unique situation, CRYOPDP as a specialist in temperature-controlled logistics, with the mission to improve people's healthcare options, had to go further and dive deeper to better understand customers perceptions of the impact of the pandemic and work with them to provide those options.With the patient always at the centre of its business and considering the huge development of decentralised clinical trials, the development and implementation of a new service such as Direct-to-Patientseems to be the most natural evolution for CRYOPDP. We have been offering this turnkey solution completely adapted to patients needs, with the same efficiency and total peace of mind, to the benefit of many customers.This model makes life a lot easier for patients and their families, as they dont have to make multiple trips to a clinic or hospital that is potentially a great distance from their home. It is also beneficial for patients who may be too ill to travel, as well as saves patients time and money.Because of these benefits, the direct-to-patient model has increased clinical trial recruitment by up to 60 percent and helped to maintain patient retention by over 95 percent. Drug developers can also gain access to a larger patient population by onboarding those who are not located near participating hospitals or clinics.Patient centricity becomes more evident with the new generation of cell and gene therapies.From day one, CRYOPDP temperature-controlled logistics solutions have always been essential to improve and save patients lives. But, when speaking about cell and gene therapies in particular, this gains an even more significant meaning.The increase in personalised medicine, advanced therapies, and improved access to healthcare in the developing world are influencing future supply chain solutions development. Cell and gene therapies are enabling the healthcare community to shift the arrow and think about patients in a whole new way.Cell and gene therapies demand rigorous and precise temperature control to ensure that the therapies maintain their viability. And maintaining temperature control calls for flawless implementation and execution.From designing the best transportation route to selecting the correct packaging, every detail is critical to keep product integrity high under all conditions, and for this to happen, the healthcare community can count on CRYOPDP specialists who can meticulously handle the entire supply chain process. We've been supporting the life sciences and healthcare communities and focused on improving patient centricity with innovative temperature-controlled logistics solutions because thinking about the patient and the outcome of our work is what moves and inspires us to be better every day.

In all the geographies of the world that we cover, around 150 countries, we produce an operational performance of 99.96%. And to deliver such operational performance, we count on our employees, the specialists around the globe, to follow our quality standards and protocols in detail, so we can deliver a quality service.

When dealing with patients lives, there is no room for errors. Its all about quality of service making sure that lifesaving samples are distributed on time, within the correct specifications and at the right temperature to ensure the patient's health is never compromised.This commitment has helped CRYOPDP to win numerous industry awards, including Best Clinical Trial Logistics Provider in APAC at the Bioprocessing Excellence Awards 2021 and Most Advanced Healthcare Solution Providers from Europe 2021 by Healthcare Insights Magazine.Our achievements are being recognised across the industry, and as we continue to improve our services for healthcare communities, it will be the end patient that benefits the most.

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The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development - Contract Pharma

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Purespring Therapeutics and SwanBio Therapeutics announce licensing agreement for use of FunSel screening platform – Yahoo Finance

Posted: July 19, 2022 at 2:29 am

Purespring Therapeutics

Purespring Therapeutics and SwanBio Therapeutics announce licensing agreement for use of FunSel screening platform

FunSel is Puresprings proprietary in-vivo gene search engine, which allows functional selection of therapeutic factors unconstrained by previous drug discovery bias

SwanBio will use FunSel to advance AAV-based gene therapies for use in neurological diseases

London, UK & Philadelphia, USA 18 July 2022Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, has entered into a collaborative licensing agreement with SwanBio Therapeutics, a gene therapy company advancing adeno-associated virus (AAV)-based therapies for inherited neurological conditions, for the use of FunSel, Puresprings proprietary gene therapy search engine.

FunSel enables a completely new way of discovering effective biological drugs, removing the narrow, target-focused bias built into traditional drug discovery. FunSel allows functional selection of the most effective factor for disease phenotype correction, starting from a library of thousands of AAV vectors that are selected directly in vivo. All factors in the FunSel library are secreted outside the cells, maximising therapeutic benefit. FunSel is gene and mechanism of action agnostic and is based on the physiology of the organ and disease in question.

Under the terms of the agreement, SwanBio will license the FunSel screening platform from Purespring to explore new targets for early-stage pipeline programs. SwanBio will have an option to develop any targets identified and to own and commercialise any molecules developed. Purespring will be entitled to milestones and royalties upon successful commercialisation. Additional financial terms are not being disclosed.

The FunSel platform has already been instrumental in the creation of Puresprings sister company Forcefield Therapeutics, a pioneer of best-in-class therapeutics to protect heart function, where FunSel was used to discover naturally occurring proteins that retain heart function and help existing cardiomyocytes protect themselves after myocardial infarction.

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Richard Francis, Chief Executive Officer of Purespring, commented: FunSel brings unique and pioneering screening capabilities and can help deliver the promise of gene therapy to non-monogenic disorders, allowing far broader patient populations to be treated than is the case with most gene therapies. This is the second example of how this gene search engine is helping to underpin a companys development strategy and demonstrates our ability to maximise the value of this platform by sharing this unique capability. SwanBio has a clear mission to develop AAV therapies for neurological disorders and its great to be able to support them and potentially others exploring new targets.

Tom Anderson, Chief Executive Officer of SwanBio, added: FunSel has the potential to enable acceleration and greater precision of candidate selection, and SwanBios application of FunSel has the potential to expand and confirm our early pipeline candidates. SwanBio is committed to advancing AAV-based therapies for the most debilitating neurological disorders and this technology promises to significantly support our ability to refine and further develop potential candidates for development.

- ENDS -

For further information, contact:

Purespring:

Richard Francis, CEOcontact@purespringtx.com+44 (0)20 3855 6324LinkedIn

Consilium Strategic Communications:

Amber Fennell, Jessica Hodgson, Genevieve Wilsonpurespring@consilium-comms.com

SwanBio Therapeutics:

Lara Furstlfurst@swanbiotx.com+1 703 946 0183

Notes to Editors

About Purespring

Purespring is the first company to treat kidney diseases by directly targeting the podocyte, a specialised kidney cell implicated in many kidney diseases, through AAV gene therapy.

Headed by former Sandoz CEO, Richard Francis, Purespring was founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases. Purespring seeks to advance gene therapies for the treatment of both monogenic and non-monogenic chronic renal diseases that are currently poorly addressed with existing treatments.

The company also has a proprietary in-vivo pipeline engine, FunSel, which is a library of all biological factors that could be candidates for gene therapy, combined with a screening method to evaluate these factors in disease models. FunSel allows Purespring to discover new gene therapy candidates across all indications, unconstrained by genetics, to find the right candidate to make the best therapy.

An initial 45 million commitment to Purespring from Syncona Ltd is enabling Purespring to progress its assets to the clinic. Synconas Chief Investment Officer, Chris Hollowood, serves as Chairman. For more information please visit: purespringtx.com and follow us on LinkedIn.

About FunSel

FunSel is a proprietary in vivo gene therapy search engine developed by Purespring Therapeutics co-founder Professor Mauro Giacca, Professor of Cardiovascular Sciences at Kings College, London.

FunSel is gene agnostic: unconstrained by genetics and therefore does not rely on knowledge of which gene is causing a disease. It allows functional selection of therapeutic factors for disease phenotype correction. FunSel contains a library of thousands of AAV vectors encoding secreted proteins.

Because it is gene agnostic, FunSel offers potential to allow the application of gene therapy to non-monogenic disorders and treat much broader patient populations.

The platform has already been instrumental in the creation of Purespring Therapeutics and sister company Forcefield Therapeutics, a pioneer of best-in-class therapeutics to protect heart function and has broad potential for researchers developing gene therapy in many disease areas. It provides Purespring with opportunities to support industry peers through partnering and potential collaborations.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTx.com.

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Purespring Therapeutics and SwanBio Therapeutics announce licensing agreement for use of FunSel screening platform - Yahoo Finance

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