StemCell Therapy

Vasi, Krista MPA; O'Donnell, Nancy MA

Usher syndrome (USH) is the most common genetic cause of combined deafness and blindness. It is a rare disease, affecting about 400,000 individuals worldwide and 20,000 to 50,000 individuals in the United States.1,2 While there is no cure for this condition, over a century of scientific discovery has resulted in tremendous advances toward the development of treatments. Despite these advances, less than one percent of people with USH is known to researchers, putting future research and clinical trials at risk. Hearing health care professionals can play a critical role in identifying children and adults with USH.

Usher syndrome was first described in 1858 by German ophthalmologist Albrecht von Graefe, who studied three siblings with deafness and progressive vision loss caused by retinitis pigmentosa (RP). In 1914, Scottish ophthalmologist Charles Howard Usher studied 69 families and identified an inherited recessive condition that caused deafness and progressive vision loss in a subset of these families.3 This syndrome was later named after Usher.

The early years of USH research were focused on identifying the different types of Usher syndrome as well as the prevalence and psychosocial aspects. Before genetic testing of this condition became available, children and adults were diagnosed phenotypically based on a history of early-onset hearing loss, progressive vision loss, and the absence or presence of vestibular involvement.

Three types and nine confirmed genetic subtypes of Usher syndrome have been identified to date (Table 14), distinguished by the severity of hearing loss, age of onset of hearing and vision loss, and the presence or absence of vestibular function.

In 1995, the first causative gene for Usher syndrome was identified. In subsequent years, additional discoveries located genes for five subtypes within USH1, three for USH2, and in 2001, the gene for USH3A.

Historically, a diagnosis of Usher syndrome was devastating, offering few options and resources. Many were told that they would be blind by a certain age, and that the syndrome is so rare they would probably never meet another person with USH. In addition, they had no knowledge of, or access to, researchers around the world who were working to understand Usher syndrome.

Early diagnosis of Usher syndrome is critical so that individuals and families can find support, make informed decisions about communication, and consider options for education, employment, and beyond. However, finding those with USH remains a challenge. The 2017 National Child Count of Children and Youth Who Are Deaf-Blind5 reported only 329 children with Usher syndrome in the United States. This number represents only a fraction of the thousands of children estimated to have USH.6 The Usher Syndrome Coalition maintains the largest international registry of individuals of all ages and types of Usher syndrome, yet that too has less than one percent of the total population registered.

Universal newborn hearing screenings have dramatically increased the number of babies identified as deaf or hard of hearing.7 Infants who fail this screening are referred to a hearing health care professional for follow-up. These professionals can play a pivotal role in recognizing the early symptoms of Usher syndrome by understanding its functional impact on a child's development and behavior. These include:

More than a century of scientific discovery has resulted in tremendous advances toward the understanding of USH and the development of treatments for this rare genetic disorder. To ensure that treatment development and clinical trials are not delayed, researchers must have access to their greatest resourcepeople living with Usher syndrome. As the first point of contact, hearing health care professionals can play a critical role in helping to identify infants, children, and adults with this condition. Together, we can help to find, educate, support, and connect the Usher community, one person at a time.

Continued here:
Building, Supporting the Usher Syndrome Community : The Hearing Journal - LWW Journals

Savannah Guthrie is recovering from a serious eye injury after being hit in the eye with a rather large toy trainthrown by her 2-year-old son.

The "Today" show co-host posted on social media and called her colleagues Wednesday to say she had suffered a torn retina when her boy, Charley, accidentally hit her in the face while playing with his train.

Torn retinas carry a high risk of retinal detachment, which can lead to permanent blindness.

"It happened last week, actually, and then I lost my vision in my right eye about 24 hours later," she told her fellow co-hosts. "Then it turned out to be kind of serious. They were afraid my retina was detached," she said.

Diagram of a torn and a detached retina. Mayo Clinic

The injurymost often occurs when something sharp scrapes or hits the eye, according to experts.The train belonging to Guthrie's son "has a really pointed edge and he threw it right at me," she said.

Medical treatment should be sought immediately.

Detachment occurs when that thin layer of tissue pulls completely away from the eye, leading to blood and other fluid building up behind the rip. The longer treatment is delayed, the stronger the risk of losing sight in that eye, according to the Mayo Clinic.

Signs of torn retina include reduced visionand the appearance of "floaters" and flashes in one's vision.

Not to be too gross, Guthrie said, but her lost vision was caused by internal bleeding building up behind the retinal tear. She has been undergoing laser treatments to try to seal the rip, she said.Laser treatments, which can be administered in a doctor's office, are the most common means of trying to reseal the torn tissue.

Doctors advised her to rest, she said, to avoid detaching the retina completely. Sudden body movements can make the tear worse.

Another form of treatment is cold therapy, or cryotherapy, a newer practice that involves applying an extremely cold probe to the outside of the eye.

The freezing temperature, like a laser, can seal leaking blood vessels and retinal tears.

Man, 71, Burned His Retina Looking at Eclipse As a Teen: 'Why Take a Chance With Your Eyes?'

Woman Recounts How Eyeball Exploded After She Was Struck by Golf Ball at Ryder Cup

Would You Eat Food That Resembles an Eyeball? A Look at America's Most Exotic Fruits

Follow this link:
'Today' Co-Host Savannah Guthrie Has Torn Retina: What to Know About the Eye Injury - Inside Edition

Exposure to polluted air is associated with an increased risk of glaucoma, a debilitating eye condition that can cause blindness, according to a study.

The findings, published in the journal Investigative Ophthalmology & Visual Science, show that people in neighbourhoods with higher amounts of fine particulate matter pollution were at least six per cent more likely to report having glaucoma than those in the least-polluted areas.

"We have found yet another reason why air pollution should be addressed as a public health priority, and that avoiding sources of air pollution could be worthwhile for eye health alongside other health concerns," said lead author Paul Foster, a professor at the University College London (UCL) in the UK.

"While we cannot confirm yet that the association is causal, we hope to continue our research to determine whether air pollution does indeed cause glaucoma, and to find out if there are any avoidance strategies that could help people reduce their exposure to air pollution to mitigate the health risks," Foster said in a statement.

Glaucoma, a neurodegenerative disease, is the leading global cause of irreversible blindness and affects over 60 million people worldwide, the researchers noted.

It most commonly results from a build-up of pressure from fluid in the eye, causing damage to the optic nerve that connects the eye to the brain, they said.

"Most risk factors for glaucoma are out of our control, such as older age or genetics. It's promising that we may have now identified a second risk factor for glaucoma, after eye pressure, that can be modified by lifestyle, treatment or policy changes," Foster said.

The findings were based on 111,370 participants of the UK Biobank study cohort, who underwent eye tests from 2006 to 2010 at sites across Britain.

The participants underwent a test to measure intraocular pressure, and a laser scan of the retina to measure thickness of their eye's macula, the central area of the retina.

The participants' data was linked to air pollution measures for their home addresses, with the researchers focusing on fine particulate matter, equal or less than 2.5 micrometres in diameter, or PM2.5.

The team found that people in the most-polluted 25 per cent of areas were at least six per cent more likely to report having glaucoma than those in the least-polluted quartile.

They were also significantly more likely to have a thinner retina, one of the changes typical of glaucoma progression, the researchers found.

Eye pressure was not associated with air pollution, which the researchers said suggests that air pollution may affect glaucoma risk through a different mechanism.

"Air pollution may be contributing to glaucoma due to the constriction of blood vessels, which ties into air pollution's links to an increased risk of heart problems," said the study's first author, Sharon Chua from UCL Institute of Ophthalmology and Moorfields Eye Hospital.

"Another possibility is that particulates may have a direct toxic effect damaging the nervous system and contributing to inflammation," Chua said.

The researchers noted that air pollution has been implicated in elevated risk of pulmonary and cardiovascular disease as well as brain conditions such as Alzheimer's disease, Parkinson's disease and stroke.

Particulate matter exposure is one of the strongest predictors of mortality among air pollutants.

The latest study adds to previous evidence that people in urban areas are 50 per cent more likely to have glaucoma than those in rural areas, suggesting that air pollution may be a key contributor to that pattern.

"We found a striking correlation between particulate matter exposure and glaucoma. Given that this was in the UK, which has relatively low particulate matter pollution on the global scale, glaucoma may be even more strongly impacted by air pollution elsewhere in the world," Foster said.

"And as we did not include indoor air pollution and workplace exposure in our analysis, the real effect may be even greater," he said.

Originally posted here:
Air pollution linked to higher risk of glaucoma: Study - Deccan Herald

Friday, Congress members Terri A. Sewell (D-Selma), Tom Reed (R-New York) and Diana DeGette (D-Colorado) introduced the Diabetic Vision Loss and Blindness Prevention Act. The legislation allows primary care physicians to perform eye tests on Medicare enrollees in an effort to encourage the early detection of diabetes-related vision loss.

Diabetic retinopathy is the most common cause of adult vision impairment in the United States and, if not found and treated early, can cause permanent vision loss, Sewell said. When it comes to our fight to improve outcomes for those living with diabetes, we need to do everything in our power to give patients the tools to stay healthy, especially for those living in rural and underserved communities without access to specialty doctors. The Diabetic Vision Loss and Blindness Prevention Act is a commonsense, bipartisan measure that will allow more Alabamians to receive critical care from their family doctors.

More than 80 percent of people living with diabetes develop diabetic retinopathy which causes vision loss. However, many people are not getting tested for the disease because Medicare will only reimburse specialty doctors, Reed said. I care about those struggling with diabetes, and want to ensure they have quick and efficient access to care. There is no fair reason for diabetics on Medicare to wait to see a specialist for a simple test their family doctor has the ability to conduct.

There are more than 30 million people in the U.S. living with some form of diabetes who, without the proper treatment, could start to lose their vision as a result, DeGette said. In addition to lowering the cost of insulin, we need to make sure that everyone with diabetes has access to the eye care they need to prevent one of the worst effects of this disease.

According to the Centers for Disease Control and Prevention, the number of Americans with diabetic retinopathy, which can cause vision loss and blindness, is expected to nearly double by 2050, from 7.7 to 14.6 million.

Under current law Medicare only reimburses specialty doctors for testing, leaving many diabetes patients without access to sight-saving vision exams. The Diabetic Vision Loss and Blindness Prevention Act reforms the reimbursement requirements for Medicare, allowing Medicare-enrolled patients to receive annual vision exams that are integral to early diagnosis and vision protection.

State Senator Jim McClendon (R-Springville) who is an optometrist told the Alabama Political Reporter: Medicare pays optometrists for any exam or visit with a medical diagnosis- diabetic retinopathy, dry eye, cataract, glaucoma and so on. Medicare does not pay any provider for routine eye exams (update glasses Rx for example).

Advertisement

Currently, primary care physicians refer patients to optometrists and ophthalmologists for testing for diabetic retinopathy.

Advertisement

Reps. Sewell and Reed are courageous leaders in Congress who are taking decisive action against the nations diabetes epidemic and the tragic toll its taking on Americas families, said Barbara L. Horn, O.D., President of the American Osteopathic Association. The Sewell-Reed bill, which AOA supports, represents an effective, efficient, and thoroughly bipartisan approach to fighting back against diabetes

Rep. Terri A. Sewell represents Alabamas Seventh Congressional District.

Read the original post:
Reps. Sewell introduces legislation to improve access to diabetic vision testing - alreporter.com

By NDUNG'U GACHANEMore by this Author

A blind prison warder at Muranga GK Prison has beaten all odds to diligently serve her country and discharge her duties as a deacon at the Anglican Church of Kenya, Diocese of Mt Kenya Central.

Ms Esther Nyawira Kamindo, who lost her eyesight in 2014 after a long battle with diabetes, visualised a bleak future and feared that her employer, the Prisons Service, would declare her redundant. She was at a crossroads.

She did not know whether to soldier on or despair in life as she could no longer perform her duties at the prison, including escorting inmates to the courts and hospitals.

Worse still, how would her husband, in-laws and friends react to her condition? She wondered.

I could not commit suicide, since Im a believer, but I lost the purpose to live. Whenever I heard that a friend or a relative died, I wished it was me. I was hopeless and could not imagine that I would catch up with life again, she told the Nation.

As fate would have it, she lost some close friends who could not tolerate a blind friend and she now had to cope with a new way of life, where friends and strangers alike discussed her in hushed tones.

From a person who used to enjoy the beauty of the world to one covered in a world of darkness, a person who could perform all household chores to someone who was dependent on others for everything, and having to cope with gossip from people who knew me before I went blind, it was not easy. It was tormenting and challenging, she adds.

But her family has remained supportive and with help from her employer, she underwent counselling and rehabilitation, learning Braille, living skills such as orientation with the surroundings, mobility and computer studies.

She accepted her situation and this kept her moving. And though tormented by the turn of events, Ms Nyawira gradually accepted that she could no longer see and had to take her studies seriously to compliment her new life as a blind warder.

In 2016, she completed her studies and went back to her work station as a counsellor, since she could no longer discharge her earlier duties.

A white cane became her companion. But even with the milestones made to readjust her life, Ms Nyawira has to live with insults from some drivers and matatu conductors, who do not understand that she cannot see when crossing the road.

Some even attempt to assault her and she has to explain that she is visually impaired.

These are just some of the things that I have to cope with as a person living with disability. When I look at my earlier life, I just feel the urge to serve the Lord.

People like me have been neglected but I was not. Some were fired; I was not; while some have isolated themselves because of stress, she said.

Ms Nyawira says her employer has been supportive, enabling her to complete theological studies and attain the position of a deacon at ACK.

I will be ordained on December 27 this year, and I will use the position to not only spread the gospel but to reach out to all those who have either lost their eyesight or a function of their body but have refused to accept themselves, she says.

I will also use myself as an example to help people understand that we are human beings too and we need acceptance despite our shortcomings.

ACK Bishop Timothy Gicere believes that by becoming a deacon, Ms Nyawira will have fulfilled her calling and that it will be a lesson that God uses us differently.

See the rest here:
Loss of sight fails to dim warder's dreams of brighter future - Daily Nation

Issued on: 27/11/2019 - 23:20Modified: 27/11/2019 - 23:18

Geneva (AFP)

Measles has killed more than 5,000 people in the Democratic Republic of Congo since January -- more than double the toll in the country's Ebola epidemic -- the World Health Organization said Wednesday.

"The DRC (measles) outbreak is the largest outbreak worldwide. It is one of the largest that we have seen," Kate O'Brien, director of the WHO's immunisation department, told reporters in Geneva.

As of November 17, the country had registered 250,270 measles cases, including 5,110 deaths, the WHO said.

DRC declared its latest measles epidemic in June, and in September the country launched an emergency vaccination campaign to counter the outbreak.

WHO said that campaign was still ongoing, but was expected to be completed by the end of the year.

O'Brien said the epidemic was still "all over the country", pointing out that most of those affected were "children and babies".

Measles is a highly-contagious disease caused by a virus that attacks mainly children. The most serious complications include blindness, brain swelling, diarrhoea, and severe respiratory infections.

The rapid spread of measles in DRC has garnered far less attention than the Ebola epidemic that has also been raging in the east of the country since August 2018. That outbreak has killed some 2,200 people.

Ian Norton, at WHO's Emergency Medical Team Unit, said the UN agency had begun training some of the Ebola teams in DRC to also manage measles cases, "because there is such a large burden".

Efforts to rein in the spread of both Ebola and measles are meanwhile being hampered by violence and unrest across the country, and especially in the east.

On Tuesday, WHO said it had moved 49 Ebola-response staff out of the eastern town of Beni as insecurity in the area surged.

Ninety-four civilians in the Beni area have been killed by armed groups since November 5, following the launch of an offensive by the country's army, according to the Congo Research Group (CRG), a not-for-profit organisation.

"The Ebola response is being hampered by the insecurity," Norton told reporters.

"The same can be said... for the measles response, not just in Beni but across the entire country," he said.

Attacks, even when not directed at medical staff or facilities, can block health workers from getting to work, and can prompt people feeling ill to stay home instead of seeking help.

Norton said the WHO was "extremely worried" by the growing insecurity, warning it had a dire "impact on the management of disease".

2019 AFP

Read the original:
More than 5,000 measles deaths in DR Congo this year: WHO - FRANCE 24

File photo: Young enthusiasts view the solar eclipse through special glasses at the Birla Planetarium in Hyderabad

On December 26, 2019, the third and final solar eclipse of the year will serve a visual treat to several parts of India between 8 am IST and last until 11:30 am IST.

This eclipse will be an annular solar eclipse a type of eclipse that occurs when the apparent diameter of the moon is smaller than that of the Sun. As the moon fails to block all of the Suns light, and only covers its centre, the Suns visible outer edges form a magnificent ring of fire in the sky. The annularity will occur between 9:24 am and 09:27 am on the east coast and 9:31 am and 9:35 am on the west coast.

Across India, the eclipse will be annular or partial depending on the location. Either way, when looking at this rare astronomical event, it is crucial to follow a list of dos and donts to enjoy this phenomenon without harming yourself.

Donts

Do not look at the Sun directly: Watching a solar eclipse without safety equipment, even for a few seconds, could be very harmful to the eyes. The Suns UV radiation can scorch and burn your retinas, and even cause permanent blindness.

Do not use films: Contrary to popular belief, colour films and medical X-ray films offer zero protection against the Suns ultraviolet rays during a solar eclipse.

Do not use regular sunglasses: Normal sunglasses, even those with UV protection, will not sufficiently protect your eyes when viewing an eclipse.

Do not use everyday optical devices: Gazing at the eclipse using binoculars, camera viewfinders, and telescopes can lead to instant and permanent blindness.

Do not use household and homemade objects: Using smoked glass, floppy disks or any other homemade solar filters should be strictly avoided.

A solar eclipse observed safely and indirectly, as the Sun's light passes through a pair of binoculars and falls on a piece of paper.

Dos

Wear eclipse glasses: A specially-made pair of eclipse glasses and eye-wear with sun filters are the best equipment to view the spectacle. If you regularly wear eyeglasses, wear the eclipse glasses on top of your regular eyeglasses.

Make your own projectors: An old-school DIY solar eclipse viewer made using a cardboard box (also known as pinhole camera) is perhaps the safest method to experience a solar eclipse, as it enables you to observe the Sun indirectly. Click here to learn how to make one. And as shown in the picture above, the Sun's reflection can also be indirectly observed by using your binoculars as projectors.

Use solar filters: If you plan on documenting the eclipse with any photo equipment, you can make use of special solar filters. However, it is best to seek expert advice from an astronomer before using the filters. Furthermore, ensure you use the type that is specifically designed for eclipses. Care should be taken, as filters can crack under the Suns magnified and focused intensity, and quickly become damaged and unsafe to use. Always check the filters before use.

Supervise your children: During an eclipse, make sure the children use the eclipse-viewing devices properly, and at no time do they directly look at the Sun with their naked eyes.

Make sure you are at the right place at the right time: Click here to find out if the annular eclipse will be visible from your area of residence, and at what time. Happy viewing!

Originally posted here:
Annular Solar Eclipse 2019: Do's and Don'ts While Watching the 'Ring of Fire' - The Weather Channel

A child elephant born with albinism has been thriving within the wild regardless of battling issues attributable to its pores and skin pigmentation.

The uncommon calf was born in April this yr in Maasai Mara, Kenya.

It was caught on digital camera by a wildlife photographer who noticed its uncommon pigmentation of pink pores and skin however its considered thriving nonetheless.

The calf was born in April earlier this yr earlier than being noticed by a wildlife photographer in Maasai Mara, Kenya

The new child with albinism has been thriving within the wild regardless of battling with intense daylight beaming down on its non-pigmented pores and skin

Mostafa Elbrolosy, a ranger who runs a safari camp, mentioned that he had heard concerning the delivery of the elephant however was stunned when he noticed the lovely calf for himself

Mostafa Elbrolosy, a ranger who runs a safari camp, mentioned that he had heard concerning the delivery of the elephant however was stunned when he noticed the lovely calf for himself.

He mentioned: It was a uncommon sighting.

Uncommon creatures are at all times essentially the most enticing for any wildlife photographer and getting the possibility to observe and it was like a dream.

As I dwell within the Maasai Mara working my cozy camp right here, I acquired a information over the radio of feminine elephant giving delivery to an albino child.

I completed my job, packed my digital camera and went searching for it with one among our guides.

We acquired the shock within the afternoon after quiet very long time of looking solely only a few folks had come to see it as nobody anticipated it to be an albino one.

I used to be very fortunate to get the possibility to see and this extraordinarily uncommon child.

Albinism is attributable to a scarcity of pigment within the pores and skin and often leads to pale dermatological layers in addition to unpigmented, pink eyes

The situation may trigger poor eyesight that would finally imply the newborn elephant suffers with blindness because it will get older

There are various elephants with non-pigmented patches of pores and skin behind their ears however true albinos can typically be rejected by their very own species resulting from their uncommon look

Mr Elbrolosy mentioned the new child calf was extraordinarily uncommon and was thriving regardless of the tough daylight which was not appropriate for it.

He was surrounded and really effectively protected by the herd, attempting to feed from the mum and stroll together with her.

He was simply eighthours outdated.

We had been thrilled to see such an incredible sight.

Albinism is attributable to a scarcity of pigment within the pores and skin and often leads topale dermatological layers in addition to unpigmented, pink eyes.

The situation may trigger poor eyesight that would finally result in blindness because the calf will get older.

There are various elephants with non-pigmented patches of pores and skin behind their ears however true albinos can typically be rejected by their very own species resulting from their uncommon look.

See the rest here:
Elephant with albinism thriving in the Kenyan wild despite problems caused by its skin pigmentation - Herald Publicist

It will be mandatory for dog owners to carry a litter bag when out with their pets in public if a new Bill introduced in the Dil on Wednesday is passed.

The legislation is aimed at a small minority of dog owners who simply do not give a damn what their dog does and have no real fear of being caught or penalised under current legislation.

Introducing the Litter Pollution (Amendment) (Dog Litter Control) Bill, Sinn Fin TD Sen Crowe said existing laws are not fit for purpose.

It is an offence for a dog owner not to pick up after their dogs faeces but our parks, footpaths, beaches and community areas are still littered with dog excrement, he said.

The problem with existing regulation is in part because a dog warden has to actually witness a dog fouling and the owner leaving the waste behind to pursue a prosecution, the Dublin South-West TD said.

The Bill is co-sponsored by his Carlow-Kilkenny Kathleen Funchion.

Mr Crowe stressed that it is not anti-dog owner legislation but was pro-health and pro- trying to eliminate the practice.

It aims to impress on owners and those in charge of dogs the importance of picking up their pets faeces when in public.

The legislation makes it an offence not to produce a suitable bag or instrument when in control of a dog.

He rejected claims by some people that it was not a serious issue and said it is a public health issue because dog waste carried disease that if contracted by humans could be life changing.

Mr Crowe referred to an illness called Toxocariasis caused by the parasitic roundworm which lives in the intestines of dogs and cats. Human contact with the eggs produced by the roundworm and excreted in a dogs faeces can cause fever, cough or wheezing, enlarged liver, rash, swollen lymph nodes and even blindness.

He added that it is particularly dangerous for children and pregnant women or those with a low immune system.

The new Bill will make it easier for wardens and An Garda Sochna to impose fines on irresponsible dog owners.

He also called for more bins for dog litter.

More:
New Bill aimed at dog owners who simply do not give a damn what their pet does - The Irish Times

Studies have found that a long lifespan may be linked to a number of factors with genetics being one of the main ones. One study from the Albert Einstein College of Medicine looked at 500 individuals who had lived to 95 or older and identified common genotypes, rather than lifestyle variables, that caused them to outlive others. For those who dont have the best genetics, fear not, studies have proven that doing this type of exercise could help.

Researchers have identified certain behaviours that can increase longevity.

Diet is of course strongly linked to longevity and it has been advised to follow a Mediterranean diet which includes plenty of fruits, vegetables, whole grains, nuts and healthy fats.

When it comes to food, the ones to avoid include processed snacks, fried foods and sugar-sweetened beverages.

Exercise can also play a big role in life longevity, in particular a certain type.

READ MORE: How to live longer: Best diet to boost life expectancy - three foods to eat

Strength in the muscles and body has also been aligned with living longer.

A study of more than a million Swedish teenage boys supports concluded that "low muscular strength in adolescents is an emerging risk factor for major causes of death in young adulthood.

Those who scored about average during initial muscular strength tests were at a 20-35 percent lower risk of early death from any cause, including cardiovascular disease.

DONT MISS

Exercise regularly

Working out is highly beneficial not only for physical health but for mental health too.

Exercise boosts everything from cardiovascular fitness to mood and energy so its no surprise it can also extend ones life.

Federal physical activity guidelines recommend aiming for at least 150 minutes of moderate or 75 minutes of vigorous aerobic activity each week, plus twice-weekly muscle-strengthening sessions.

Benefits of team sports

Opting for a more vigorous workout, leading health experts recommend partaking in more team sports such as tennis and football.

Its believed that team sports help boost longevity as they encourage social interaction as well as exercise.

A study published in Mayo Clinic Proceedings found that the social interaction one gets from working out with someone else such as participating in team sports can actually be more beneficial than working out alone, adding years to ones life.

Study co-author, Dr James OKeefe, a cardiologist at Saint Lukes Mid America Heart Institute said: If youre interested in exercising for health and longevity and well-being, perhaps the most important feature of your exercise regimen is that it should involve a playdate.

Professor of psychology and logopedics at the University of Helsinki, Finland added: It would be important to maintain existing relationships by meeting family members or friends face-to-face and exercising together.

If your social network isnt where you want it to be, consider looking for recreational sport leagues you can join or group fitness classes where you might meet some new, friendly faces.

See more here:
How to live longer: This type of exercise found to be best for increasing life expectancy - Express

JERSEY CITY, N.J.--(BUSINESS WIRE)--

Ceremony to host Bonnie Bassler, molecular biologist and microbe fighter; Robert J. Hariri, stem cell and human longevity expert; and David Rosenberg, world leader in urban vertical farming

Plus a special honoree from California whom LSC is feting because hes a tech badass: AI giant Sebastian Thrun, the godfather of the self-driving car

New Jersey is home to some of the worlds most accomplished innovators in applied science. Three of them who are pioneering research and solutions in antibacterial therapies, genetics, human life extension, and food production are being honored by Liberty Science Center at its inaugural The Genius of NJ celebration on Monday, December 2.

The celebration starts at 5:30 pm with cocktails and unique technology demonstrations: a full-body 3D scanner from Lenscloud that can scan a person in half a second with 120 cameras and create a realistic 3D avatar; bomb-disposing robots and an autonomous fighting robot from Picatinny Arsenal; and Flyer, a personal aerial vehicle from Kitty Hawk, headquartered in Mountain View, CA.

The New Jersey honorees are Bonnie Bassler, Chair of Molecular Biology at Princeton University, who is developing novel antimicrobial therapies to render pathogenic bacteria harmless; Dr. Robert J. Hariri, Chairman, Founder & CEO of Celularity, Inc. who is pioneering the use of stem cells to cure disease and slow aging; and David Rosenberg, CEO and Co-Founder of AeroFarms, the worlds leader in mass-scale vertical indoor farming.

Our inaugural Genius of NJ Award Winners represent the best this state and the world have to offer in harnessing science for the betterment of humanity, said Liberty Science Center President and CEO Paul Hoffman. Each is using his or her exceptional intellect and creative abilities to disrupt and innovate both in their respective fields and in their commitment to making the world healthier and safer.

Bonnie Bassler is the Squibb Professor of Molecular Biology and Chair of the Department of Molecular Biology at Princeton University, as well as a Howard Hughes Medical Institute Investigator. Professor Bassler deciphered the chemical language bacteria cells use to communicate by studying a harmless marine bacterium called Vibrio fischeri, known to bioluminesce, or make light, like fireflies do. She is a winner of the MacArthur Genius Grant and is now developing therapies that disrupt communication among harmful bacteria and strengthen communication among helpful bacteria. At a time when an increasing number of bacteria are resistant to traditional kinds of antibiotics, Dr. Bassler offers a promising new approach to antimicrobial therapy.

The Chairman, Founder and CEO of Celularity, Inc., in Warren, NJ, and Co-Founder and Vice Chairman of Human Longevity, Inc., Dr. Robert Hariri is the quintessential renaissance man. Hes a neurosurgeon, a medical researcher, and a serial entrepreneur in two technology sectors: aerospace and biomedicine. Dr. Hariri has advised the Vatican on genetics, and in 2018, Pope Francis bestowed on him the Pontifical Key Award for Innovation. Dr. Hariris path to discovering that the placenta, a temporary organ discarded after birth, was a potent source of stem cells began in the 80s when he viewed a first trimester ultrasound of his oldest daughter and wondered why the placenta was so large. Today Dr. Hariri is working to use placental stem cells to cure disease, slow aging, and augment healthy human lifespan.

Prominent entrepreneur David Rosenberg, CEO and Co-Founder of AeroFarms, set out to reinvent one of the most basic aspects of food production, farming. AeroFarms has grown 800 species of plants indoors and can grow them 365 days a year without sun or soil, achieving yields 130 times greater than conventional farming. His system uses 95 percent less water than field farming and no pesticides, herbicides, or fungicides. Rosenbergs adoption of cutting-edge technology has been a cornerstone of AeroFarms, which set up its first indoor vertical farms in abandoned warehouses in Newark. He employs plant biologists, microbiologists, geneticists, systems engineers, and data scientists. AeroFarms innovations in indoor vertical farming have improved not just plant yields but also taste, texture, nutritional density, and shelf life.

Story continues

Additionally, LSC will honor non-New Jersian Sebastian Thrun, CEO of Kitty Hawk, a company spun off from a Google moonshot effort to free the world from traffic. Kitty Hawk is developing all-electric, vertical take-off flying machines for everyday use. Known as the godfather of self-driving cars, as a Stanford professor in 2005, Thrun led a team that won the $2-million Defense Department Grand Challenge to build an autonomous vehicle which drove itself unassisted on a 132-mile course across the Mojave Desert. His winning entry, Stanley, is now on display at the Smithsonian in Washington, DC. While at Stanford, in 2011 he and colleague Peter Norvig offered their Introduction to Artificial Intelligence course online to anyone, for free. Over 160,000 students in more than 190 countries enrolled! The MOOC (which stands for Massive Open Online Course) was born, and Thrun founded the online education company Udacity, with the goal of democratizing education. Thrun relinquished his tenured Stanford professorship to join Google and founded the companys semi-secret R&D division called Google X (now called simply X) to develop breakthrough technologies, such as self-driving cars, that make the world a radically better place.

Ticket prices for The Genius of NJ start at $750 per guest with options for table sponsorship from $12,500 to $50,000. For more details, please visit The Genius of NJ online. All proceeds from this event will support LSCs mission to inspire the next generation of scientists and engineers.

About Liberty Science Center

Liberty Science Center (LSC.org) is a 300,000-square-foot nonprofit learning center located in Liberty State Park on the Jersey City bank of the Hudson near the Statue of Liberty. Dedicated to inspiring the next generation of scientists and engineers and bringing the power, promise, and pure fun of science and technology to learners of all ages, Liberty Science Center houses the largest planetarium in the Western Hemisphere, 12 museum exhibition halls, a live animal collection with 110 species, giant aquariums, a 3D theater, live simulcast surgeries, a tornado-force wind simulator, K-12 classrooms and labs, and teacher-development programs. More than 250,000 students visit the Science Center each year, and tens of thousands more participate in the Centers off-site and online programs. Welcoming more than 750,000 visitors annually, LSC is the largest interactive science center in the NYC-NJ metropolitan area.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191125005713/en/

See the original post here:
Liberty Science Centers Inaugural Genius of New Jersey to Honor Innovators Who Make the State a World Leader in Cutting-Edge Applied Science - Yahoo...

MIT Provost Martin A. Schmidt said sharing the risk among several institutions will not only make possible work that would be difficult for a single institution to tackle, it will also encourage collaboration that accelerates the process of moving discoveries from lab to patient.

MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use, Schmidt said. A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvards lead in convening exploration of this opportunity for the Commonwealth.

Richard McCullough, Harvards vice provost for research and professor of materials science and engineering, who also helped lead the project, said although the centers activity will revolve around science and manufacturing, its true focus will be on patients.

The centers overarching goal will be improving patient care, McCullough said. This would occur both by speeding access to the essential, modified cells that patients in clinical trials await, and by fostering discoveries through collaborations within the centers innovation space. The aim is that discoveries result in whole new treatments or improved application of existing treatments to provide relief to a wider universe of patients.

Organized as a private nonprofit, the center will be supported by more than $50 million pledged by its partners. It will be staffed by a team of at least 40, experienced in the latest cell-manufacturing techniques and trained in the use of the latest equipment. Among its goals is disseminating badly needed skills into the Boston life-sciences workforce.

We have to be sure that we are constantly feeding the industry with talented people who know the right things, so personally, I am very excited about education programs, Ligner said. Initiatives like [this center] are essential to advancing the industry because they help organizations build on one anothers advances. For example, the full potential of cell and gene therapies will only be realized if we collaborate to address challenges, such as manufacturing, improving access, accelerating innovation, tackling cost issues, and then sharing our learnings.

The new center emerged from conversations with state officials, including Gov. Charlie Baker and Attorney General Maura Healey, and industry sector leaders about ways to bolster Massachusetts preeminence in life science research and medical innovation. Those conversations sparked a two-year consultation process at the invitation of Garber and Harvard Corporation Senior Fellow Bill Lee, that was coordinated with state officials and included representatives from industry, academia, venture capital, area hospitals, and government.

Cell and gene therapies have the potential to revolutionize the global health system. Recently, in Sweden, the first patient received cell therapy outside of a clinical trial. Its the start of an incredible time in the industry and in human health.

Emmanuel Ligner, president and chief executive of GE Healthcare Life Sciences

Called the Massachusetts Life Sciences Strategies Group, members reached out to regional experts beginning in 2017to discover what fields they considered most important and how best to support them. Cell and gene therapy rose to the top because of the considerable excitement generated by activity already going on, its potential to help patients, and its high potential for future growth and innovation. Also important were the opportunities to spread the high cost of these technologies across multiple institutions and, while so doing, capture the collaborative power of housing each player in the development chain within a single facility.

The centers board of directors will be comprised of Harvard, MIT, and industry partners Fujifilm, Alexandria Real Estate Equities, and GE Healthcare Life Sciences. Other members will include Harvard-affiliated teaching hospitals Massachusetts General Hospital, Brigham and Womens Hospital, Beth Israel Deaconess Medical Center, Boston Childrens Hospital, and the Dana-Farber Cancer Institute; as well as the Commonwealth of Massachusetts and life-sciences company MilliporeSigma.

When you look at the constellation of players coming together, you really have the best universities and the best teaching hospitals and the best corporate players all supporting it, McGuire said, which I think is a great opportunity.

The facility intends to provide researchers and emerging companies outside the consortium with access to excess material, though organizers said they expect it to be in high demand by center partners.

The centers boost to the areas cell and gene therapy endeavors comes early enough that it should help maintain leadership over places like California and China, which have made clear their interest in life-science research, McGuire said.

I think getting this early mover advantage is going to be huge [in] developing the technology and the know-how and, ultimately, the intellectual property around it, McGuire said.

For Sharpe, the ultimate payoff will come from using cancer immunotherapys checkpoint blockade and other cell and gene therapies to save and improve lives.

We are seeing long-term benefits in some patients whove received checkpoint blockade, Sharpe said. There are patients who are more than a decade out and are melanoma-free. I think that it really has transformed patient care, quality of life, and longevity. So Im optimistic that the more we learn, the more were going to be able to do to help patients.

Sign up for daily emails to get the latest Harvardnews.

Go here to read the rest:
Partnership aims to accelerate cell and gene therapy - Harvard Gazette

Promising preliminary data from one of the first human trials testing the safety and efficacy of a CRISPR gene therapy has just been revealed. Although it is too early to evaluate long-term effects, the initial reports are impressively successful for two patients with severe genetic blood diseases.

Until February of this year, when pharmaceutical companies CRISPR Therapeutics and Vertex began a large global trial into a treatment called CTX001, no human outside of China had been officially treated with a CRISPR-based gene editing therapy.

CTX001 was developed to treat two types of inherited blood disease, beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene and the treatment involves engineering a patient's stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.

The newly announced data from the first two patients treated with CTX001 is nothing short of extraordinary. The first patient was treated with the CRISPR therapy at the beginning of 2019 for transfusion-dependent beta-thalassemia. The patients illness was so severe they required around 16 blood transfusions every year. Nine months after the single CTX001 treatment the patient was completely independent of the need for blood transfusions and their total hemoglobin levels were near normal.

The second patient, treated for sickle cell disease, demonstrated similar remarkable responses to the one-off gene therapy treatment. Four months after the CTX001 infusion the patients total hemoglobin levels had returned to normal and many of the disease symptoms had disappeared.

We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001, says Samarth Kulkarni, CEO of CRISPR Therapeutics. These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention.

Both patients did suffer from a small number of serious adverse events following the CTX001 treatment, however, the researchers conducting the trial are confident none of these side effects were related to the gene therapy. Instead, these side effects seem primarily related to a pre-CTX001 treatment involving the elimination of pre-existing mutated bone marrow cells to enable the healthy CRISPR-edited cells to reproduce.

It is certainly very early days for the research, and a number of patients are yet to be enrolled and treated in this current trial. Both the beta-thalassemia and sickle cell disease trials plan to enroll up to 45 patients, with a two-year follow-up planned to evaluate safety and efficacy. The Phase 1/2 open-label trials precede larger Phase 3 trials required for ultimate market approval, so these new therapies are still at least a decade away from clinical implementation.

Still, these initial results are as positive as one could hope for at this stage, establishing CRISPR gene editing as having exciting curative potential in human subjects. Whether the treatment holds for extended periods of time, and demonstrates longer safety profiles is yet to be determined. But, these early results are leaving researchers cautiously optimistic.

The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, says CEO of Vertex, Jeffrey Leiden. While the data are exciting, we are still in the early phase of this clinical program.

Source: CRISPR Therapeutics

Read more from the original source:
Encouraging early results from first human CRISPR gene therapy trials - New Atlas

CAMBRIDGE, Mass. Gene therapy pioneer Dr. James Wilson is disappointed by the progress in his field and expects current therapies and technologies to be soon surpassed by new approaches.

In five years, when we look back on the way were executing on gene therapy now, were going to realize that things are going to be very different, Wilson said at the STAT Summit on Thursday. The way in which were going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which its being evaluated in the clinic now.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!

STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis.Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

See the rest here:
Gene therapy pioneer: Field is behind, and delivery tech is 'embarrassing' - STAT

Thanks to a $62 billion acquisition of Shire, Takeda is one of the world's largest developers of rare disease drugs.

Despite that, the 238-year-old Japanese pharmaceutical company lacks any mid- or late-stage cell or gene therapies, two technologies that figure to play a large role in how many rare cancers and inherited diseases will eventually be treated.

It's a mismatch Takedais putting substantial effort into addressing. Last week, executives made cell and gene therapy a notable focus of the company's first R&D day since closing its Shire deal.

"We have a world-class gene therapy platform," Dan Curran, head of Takeda's rare disease therapeutic area unit, told investors and Wall Street analysts gathered in New York city.

"We intend to build on that over the next five years. Because as we look to lead in the second half of [next]decade, we believe patients will demand and we can deliver transformative and curative therapies to patients globally."

But right now that's just an ambition. While Takedahas begun to explore how it can improve on current gene therapies, its candidates are early stage and lag their would-be competitors.

"Our heme A program we're behind. Our heme B program we're behind," admitted Curran in an interview. "But we're behind the first generation and when has there only been one generation of anything?"

Takeda's hemophilia A program is currently in Phase 1, with the hemophilia B candidate about to join it in human testing well back from leaders BioMarin Pharmaceutical, Spark Therapeutics and SangamoTherapeutics in hemophilia A and UniQure in hemophilia B.

Curran laid out three priorities for Takeda'spush: exploring whether gene therapy, typically pitched as a one-time treatment, can be re-dosed; lowering the doses currently used for first-generation therapies; and developing alternative gene delivery vehicles than the adeno-associatedand lentiviralvectors that are predominant today.

"We need to figure out how to re-dose AAVvectors if we want to provide functional cures for patients for the rest of their lives."

How long a gene therapy's benefit lasts is a critical question. In theory, it could last decades or potentially for life, depending on the treatment's target.

But clinical evidence presented to date suggests that benefit for some therapies could wane over time. BioMarin, for example, presented data this year that it argued is proof its gene therapy could raise Factor VIII expression levels in patients with hemophilia A above the threshold for mild disease for at least eight years a long time, to be sure, but not life-long.

Still, it's an unusual objective. Much of gene therapy's promise lies in the potential for it to be given just once and still deliver lasting benefits. And the therapies that have reached market most notably Spark Therapeutics' Luxturna, Novartis' Zolgensma and Bluebird bio's Zynteglo are among the most expensive drugs to ever reach market. Were a gene therapy to be re-dosed, the current value proposition those drugmakers describe would need to be re-evaluated.

Curran recognizes that bringing down costs substantially will be essential to any attempt to advance a multi-use gene therapy. But Takeda might have an advantage. In buying Shire, the pharma inherited a viral vector manufacturing plant, originally built by Baxalta, that Curran calls the company's "best kept secret."

"It's an enormous competitive advantage," he said, adding that Takeda believes it's among the industry's top three facilities by production capacity. "Roche trying to acquire Spark, Novartis and AveXis a significant component of value of those transactions was that these companies had actually invested in manufacturing capabilities."

Curran emphasized that Takeda's ambitions in gene therapy will require it to partner with academic leaders in the field, a playbook that it's followed over the past three years as it's worked to expand into cell therapy.

"In the cell space, there's more innovation you can bring up into proof of principle milestones in academia," said Andy Plump,Takeda'shead of R&D, in an interview.

"An academic can manipulate a cell, but it's very hard in an academic setting to optimize a small molecule," he added. "This is a space where Novartis, and now we, have been quite successful in creating those relationships."

Takeda has put partnerships in place with Japan's Center for iPS Cell Research and Application, GammaDelta, Noile-Immune Biotech, Memorial Sloan Kettering Cancer Center and, just this month, The University of Texas MD Anderson Cancer Center.

That last collaboration gives Takeda access to a chimeric antigen receptor-directed natural killer, or NK, cell therapy.The drugmaker believes NK cells could offer advantages over the T cells modified to create the currently available cell therapies Kymriah and Yescarta.

Most notably, MD Anderson's approach uses NK cells isolated from umbilical cord blood, rather than extracting T cells from each individual patient a time-consuming and expensive process that has complicated the market launch of Kymriah and Yescarta. Cord blood-derived NK cells are designed to be allogeneic, or administered "off the shelf."

Additionally, CAR NK cells haven't been associated (yet) with cytokine release syndrome or neurotoxicity, two significant side effects often associated with CAR-T cell therapies. That could help Takeda position its cell therapies as an outpatient option.

"Even if we were a company that entered a little bit later into the immuno-oncology space, we've very much tried to turn this into an advantage," said Chris Arendt, head of Takeda's oncology drug discovery unit, at the company's event.

"We believe we have a chance to establish a leadership position rather than jumping on the bandwagon and being a follower."

While Takeda's choice to pursue NK cell therapy stands out, its choice of target does not. TAK-007, a drug candidate from MD Anderson that is now Takeda's lead cell therapy program, is aimed at a cell surface protein called CD19 that's found in leukemias and lymphomas.

Both Yescarta and Kymriah target CD19, and a recent count by the Cancer Research Institute tracked 181 cell therapy projects aimed at the antigen.

Takeda is planning to advance TAK-007 into pivotal studies in two types of lymphoma and chronic lymphocytic leukemia by 2021, with a potential filing for approval in 2023.

By then, Kymriah and Yescarta will have been on the market for six years and current bottlenecks in cell therapy treatment could be solved, helping both Takeda's potential entry as well as the host of competitors it will likely face.

Next year will be a test of how productive Takeda'scell therapy unit can be. In addition to TAK-007, the pharmaexpects to have four other CAR-T and gamma delta cell therapies in the clinic, two of which will target solid tumors.

Cell and gene therapy are part of what Takeda calls its "second wave" of R&D projects, a group of early-stage drugs and programs that it sees as progressing to regulatory stages by 2025 or later.

In the nearer term, the drugmakeris advancing a "first wave" of clinical candidates that it told investors will deliver 14 new molecular entities by 2024. Five of those will come in rare disease, with the others spread across oncology, neuroscience, gastro-enterology and vaccines.

"We think the cascade of news coming forward on these programs will transform how people view Takeda," Curran said.

More importantly to the investors gathered in New York, Takeda expects these experimental drugs will eventually earn $10 billion in peak annual sales, which would represent a sizable addition to a business that generated $30 billion in sales last year.

Excerpt from:
Takeda sees cell, gene therapy in its future. Is it too late? - BioPharma Dive

MANHATTAN, Kan., Nov. 21, 2019 /PRNewswire/ -- Libella Gene Therapeutics, LLC("Libella") announces an institutional review board (IRB)-approved pay-to-play clinical trial in Colombia (South America) using gene therapy that aims to treat and ultimately cure aging. This could lead to Libella offering the world's only treatment to cure and reverse aging by 20 years.

Under Libella's pay-to-play model, trial participants will be enrolled in their country of origin after paying$1 million. Participants will travel to Colombia to sign their informed consent and to receive the Libella gene therapy under a strictly controlled hospital environment.

Traditionally, aging has been viewed as a natural process. This view has shifted, and now scientists believe that aging should be seen as a disease. The research in this field has led to the belief that the kingpin of aging in humans is the shortening of our telomeres.

Telomeres are the body's biological clock. Every time a cell divides, telomeres shorten, and our cells become less efficient at dividing again. This is why we age. A significant number of scientific peer-reviewed studies have confirmed this. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

Bill Andrews, Ph.D., Libella's Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrew's gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libella's clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

Libella's clinical trial has been posted at the United States National Library of Medicine (NLM)'s clinicaltrials.gov database. Libella is the world's first and only gene therapy company with a clinical trial posted at clinicaltrials.gov that aims to reverse the condition of aging.

On why they decided to conduct its project outside the United States, Libella's President, Dr. Jeff Mathis, said, "Traditional clinical trials in the U.S. can take years and millions, or even billions,of dollars. The research and techniques that have been proven to work are ready now. We believe we have the scientist, the technology, the physicians, and the lab partners that are necessary to get this trial done faster and at a lower cost in Colombia."

Media Contact:Osvaldo R. Martinez-ClarkPhone: +1 (786) 471-7814Email: ozclark@libellagt.com

Related Files

curing_aging_booklet.pdf

Related Images

william-bill-andrews-ph-d.jpg William (Bill) Andrews, Ph.D. Dr. Bill Andrews is a scientist who has spent his entire life trying to defeat the processes that cause us to age. He has been featured in Popular Science, The Today Show, and numerous documentaries on the topic of life extension including The Immortalists documentary.

Related Links

Dr. Bill Andrews speech at RAADfest 2018 (Sept 21, San Diego, CA)

bioaccess: Libella's CRO partner in Colombia.

SOURCE Libella Gene Therapeutics, LLC

Home1

The rest is here:
Breakthrough Gene Therapy Clinical Trial is the World's First That Aims to Reverse 20 Years of Aging in Humans - PRNewswire

Subscribe to The Point, arriving in your inbox Mondaythrough Friday at 8 a.m.

When its not working and we keep getting the same outcomes, something will change and it will be an urgent matter. The Alachua County School Board is starting to put together bold plans for closing the achievement gap between the districts black and white students. (WUFT News)

Global company Thermo Fisher Scientific has concluded its $6 million expansion at Progress Park in Alachua. One of its vice presidents celebrated that progress Friday, saying, Ive been in this industry for over 20 years in gene therapy, and over the last three years, its completely transformed. (WUFT News)

Marketplace visited Gainesville to see a University of Florida professors efforts to improve the flavor of tomatoes.

State Sen. Keith Perry is taking an interest in the problem of police arresting people with epilepsy during their seizures. (WTSP)

The first two Democrats vying to replace state Rep. Clovis Watson, who is term-limited from running again next November, have filed paperwork during the past month or so. Watson now intends to unseat Alachua County Sheriff Sadie Darnell. (WUFT News, News Service of Florida)

UFs fall semester is wrapping up in the next few weeks, but as it does so, theres a racial and political mess simmering that on Friday afternoon prompted a campuswide email from a vice president. (The Alligator)

One of Micanopys longtime religious leaders is stepping down after 37 years. Les Singletons final service as vicar took place yesterday at Church of the Mediator. (WCJB)

Three years ago, it seemed Amtrak was on a path to returning to serving parts of north Florida. The process of getting passenger trains going again between Pensacola and Jacksonville has moved extremely slowly. (WUFT News, Pensacola News Journal)

A slice of old Florida has been preserved at the Orange Lake Overlook at a cost of $1.3 million. Another piece of local Florida natural history was celebrated this weekend at Little Orange Creek. (Ocala Star-Banner, WUFT News)

Correction from Fridays edition, which had the incorrect title of Ibram X. Kendis latest book. It is called How to Be an Antiracist.

Theres no denying the importance of a good education.

Millhopper Montessori is one of only two area private schools accredited by FCIS, due in part to our highly-credentialed teachers and STEAM-enhanced curriculum. Millhoppers unique methods allow each preschool through middle school student to explore and learn, all in a safe and secure environment. Millhopper students develop into poised, compassionate and creative leaders who are prepared for the next level. Call 352-375-6773 or visit millhopper.com today to schedule your tour.

WFSU put together a helpful guide on how state Supreme Court justices come to hold that position. Its particularly timely with the governor set to make two more selections.

USA Today tracked a man who in the 1990s was to face trial on rape charges in Vermont but who has been living in Florida without consequence for more than two decades.

CBS spotlights the role Florida played in the 2016 election hacking by Russian operatives, including what happened within Annette Taddeos failed run for the U.S. House.

Not everyone is on board with recreational marijuana in Florida, and that group now has a political action committee. (BayNews9)

Being a prisoner (of high water) in Key Largo is not that bad, but it is reality, a resident there tells the New York Times. Its report on King Tide flooding is worth your time.

Whats west of Key West? The Dry Tortugas, and they have a fascinating 500-year history. (Florida Memory Blog)

Heres a helpful guide on some of the states worst invasive plant species. (Florida Today)

World: Pope Francis, In Visit To Hiroshima, Says Possession Of Nuclear Weapons Is Immoral

World: Anti-Doping Agency Cites Russian Non-Compliance With Olympic Testing Procedures

National: Navy Secretary Richard V. Spencer Forced Out Amid Controversy Over SEAL Case

National: How Some Sex Abuse Victims Are Finding Different Ways To Sue Clergy In Pennsylvania

Politics: How To Have Constructive Conversations At A Divisive Thanksgiving

Politics: Schiff: Impeachment Report Now Being Written, But More Hearings Still Possible

Science: Young Researchers Feel Excitement And Sadness To See Arctic Ice That May Disappear

Books: The Survivors Author On The Inheritance Of Holocaust Trauma

Education: Economists Say Forgiving Student Debt Would Boost Economy

See more here:
The Point, Nov. 25, 2019: Gene Therapy Work Gets $6 Million Home In Alachua - WUFT

NEW YORK--(BUSINESS WIRE)--IVERIC bio, Inc. (NASDAQ: ISEE) announced today the appointment of Guangping Gao, PhD, as Chief Strategist, Gene Therapy. Dr. Gao brings over 30 years of scientific research experience in gene-based treatments. As one of the worlds leading gene therapy experts, Dr. Gaos highly distinguished career includes major contributions to the development of adeno-associated virus (AAV) gene delivery technology. Dr. Gao is the current President of the American Society of Gene and Cell Therapy (ASGCT). In his advisory role, Dr. Gao will help shape IVERIC bios gene therapy strategy going forward.

On behalf of the Board of Directors and management team at IVERIC bio, we are extremely excited to welcome Dr. Gao to IVERIC bio, stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. Our goal at IVERIC bio is to become a leading retina company with a diversified portfolio addressing significant unmet medical needs in large market age-related retinal indications and inherited retinal diseases by utilizing both therapeutics and gene therapies. Dr. Gaos extensive background in gene therapy will be instrumental in shaping the direction of our gene therapy portfolio. It is a true privilege to work with him closely and benefit from his extensive expertise during these exciting times at our company.

I am delighted to have the opportunity to be a part of the IVERIC bio team, stated Dr. Gao. I am impressed with the progress that IVERIC bio had made with their diverse pipeline of novel gene therapy solutions to treat orphan inherited retinal diseases (IRDs). I look forward to working closely with the team to help build and maximize the potential of the Companys gene therapy programs.

Dr. Gao is a world-renowned scientist and researcher who is a pioneer in AAV gene delivery technology, stated Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio. We believe that the combination of Dr. Gaos expertise in gene therapy and our knowledge and experience in drug development for retinal diseases will help set the stage for the proficient execution of our gene therapy strategy, advancing it to the next stage of evolution. We will also continue to move forward our Zimura, complement C5 inhibitor, programs as expeditiously as possible.

About Guangping Gao, PhD

Dr. Gao is the Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research, at the University of Massachusetts Medical School; an elected fellow of both the US National Academy of Inventors (NAI) and the American Academy of Microbiology; and the current President of the American Society of Gene and Cell Therapy.

Dr. Gao is an internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, providing technology to enable potential treatments for many currently untreatable human diseases. For nearly 30 years of his scientific research career, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy for rare genetic diseases, with research encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, and viral vector manufacturing for preclinical and clinical gene therapy applications, as well as development of technology platforms for novel approaches for human gene therapy.

Dr. Gao has published 267 research papers, 6 book chapters, and 5 edited books and serves as Editor of Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series, Associate Editor of Signal Transduction and Targeted Therapy, and on the editorial boards of several other gene therapy and virology journals. Dr. Gao holds 135 patents with 239 additional patent applications pending. Dr. Gaos inventions have been licensed to and are currently in development by over ten pharmaceutical companies. Recently, Dr. Gao was ranked as #4 on Nature Biotechnologys list of the Worlds Top 20 Translational Researchers for 2017.

About IVERIC bio

IVERIC bio is a biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. Vision is Our Mission. For more information on the Company please visit http://www.ivericbio.com.

IVERIC bio Forward-looking Statements

Any statements in this press release about the Companys future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Companys strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words anticipate, believe, estimate, expect, intend, goal, may, might, plan, predict, project, target, potential, will, would, could, should, continue, and similar expressions. In this press release, the Companys forward looking statements include statements about the implementation of its strategic plan to seek to address significant unmet medical needs in large market age-related retinal indications and inherited retinal diseases by utilizing both therapeutics and gene therapies, the timing, progress and results of clinical trials and other research and development activities, including manufacturing activities, the potential utility of its product candidates and the potential for its business development strategy. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the initiation and the conduct and design of research programs and clinical trials, establishment of manufacturing capabilities, availability of data from these programs, reliance on university collaborators and other third parties, expectations for regulatory matters, need for additional financing and negotiation and consummation of business development transactions and other factors discussed in the Risk Factors section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Companys views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. 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 except as required by law.

ISEE-G

Read the original post:
IVERIC bio Appoints Guangping Gao, PhD, Internationally Recognized Gene Therapy Pioneer as Chief Strategist, Gene Therapy - Business Wire

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A.

The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly known as BMN 270. Administered as a single infusion, the therapy uses adeno-associated virus (AAV) vectors to deliver a functional copy of clotting factor VIII, the protein that is missing in people with hemophilia A.

An ongoing Phase 3 trial, GENEr8-1 (NCT03370913), is investigating the treatments safety and efficacy, and is still recruiting adult patients. Go here for more information on trials locations and here for eligibility criteria.

The EMA had previously given valoctocogene roxaparvovec the designation of priority medicines, or PRIME, in 2017. Now, the potential therapy has been granted accelerated assessment, which may potentially shorten its MAA review process from 210 to 150 days.

Accelerated assessment is given by the EMAs Committee for Medicinal Products for Human Use and Committee for Advanced Therapies to innovative medications that are of major interest to public health. This endorsement is meant to speed up the review process of eligible medications, but does not impact the committees decision to recommend their approval.

BioMarins MAA submission was based on updated three-year data from a Phase 1/2 study (NCT02576795)and on an interim analysis of the ongoing Phase 3 GENEr8-1 trial (NCT03370913), which is still recruiting an anticipated 130 patients from 73 sites around the world to test the dose of 6e13 vg/kg (vector genomes per kilogram). Another Phase 3 trial, the GENEr8-2 (NCT03392974) study, is also ongoing and testing a lower dose (4e13 vg/kg).

Three-year data from the Phase 1/2 trial showed that a single administration of valoctocogene roxaparvovec at the higher dose markedly reduced bleeding episodes and the need for factor VIII infusions in a small group of adults with severe hemophilia A. Specifically, there was a 96% reduction in both the mean ABR (annualized bleed rate) and the mean factor VIII usage over the three years.

The levels of clotting factor VIII remained stable over the course of three years following treatment.

Valoctocogene roxaparvovec was generally well-tolerated by patients. None of the participants developed inhibitors to factor VIII, and none withdrew from the study due to adverse events.

We are grateful to the study participants, who have made this progress possible in the span of approximately four years since the first participant was enrolled in the clinical program, Hank Fuchs, MD, president of BioMarins global research and development, said in a press release.

We are very pleased with the level of engagement we have had with global health authorities, as it aligns with our belief that gene therapy represents the next wave of innovation and potentially could be a meaningful advancement for treating people with severe hemophilia A, Fuchs said.

Valoctocogene roxaparvovec will be the first gene therapy for hemophilia whose MAA will be reviewed by health authorities for potential approval in the E.U. BioMarin is expecting the EMA to start reviewing its application in January 2020 and said it will provide an update at that time.

In the meantime, the company is planning to submit a biologics license application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration (FDA) by the end of the year. The investigational treatment has been given a breakthrough therapy designation by the FDA, as well asorphan drug status from both the FDA and EMA.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 121

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Read more here:
For Hemophilia A, BioMarin Seeks Approval of Its Gene Therapy in Europe - Hemophilia News Today

The gene therapy field made significant strides this year, and 2020 looks set to bring further advances, particularly as a three-way race to develop a hemophilia A gene therapy progresses.

BioMarin Pharmaceutical, Spark Therapeutics and Sangamo Therapeutics are all developing gene therapies for hemophilia A, the inherited disorder characterized by frequent bleeding episodes due to the lack of an essential blood-clotting protein.

While drug candidates from all three biotechs are currently in clinical testing, each is in a distinctly different spot as the companies head into 2020. Here's an overview of how the hemophilia gene therapy space is shaping up and what to watch for over the next 12 months.

BioMarin is well-positioned to be first to market with a gene therapy known as valrox, short for valoctocogene roxaparvovec.

The San Rafael, California-based biotech reported interim Phase 3 results from 16 patients in May, and stated it would file for accelerated approval with the Food and Drug Administration by year end. Since then, BioMarin executives have reiterated that regulatory timeline.

Yet even as BioMarin nears an FDA submission, clinical questions still surround valrox.

Interim Phase 3 data fell short of an earlier trial, leading company executives to blame a difference in timing of steroid use. In the prior study, steroids were typically given to study participants three weeks after infusion, while the Phase 3 trial prescribed steroid treatment after 10 weeks.

And three-year follow-up data from that Phase 1/2 trial showed patient levels of the Factor VIII clotting protein declined over time, raising questions on the product's long-term durability.

That sets up 2020 as the potential year for valrox's approval and launch, which could make it the third or fourth gene therapy for an inherited disease to reach market.

If that occurs, BioMarin will face an important decision on setting valrox's price.Unlike Novartis' Zolgensma, a gene therapy for spinal muscular atrophy that launched this year with a $2.1 million price, hemophilia patients have a range of existing treatments that are used to manage and control their disease.

Particularly if the therapy's price falls in the seven figures, payers could very well push back and negotiate. Concepts such as installment payment plans or outcome-based arrangements have floated as options around by gene therapy companies but remain incremental changes to a system still geared toward chronic therapy.

BioMarin also plans to present four-year follow-up data from the Phase 1/2 study in mid-2020 and Phase 3 data on reducing annual bleed rates in the fourth quarter of 2020 or first quarter of 2021.

At least publicly, Spark has gone quiet on how it's hemophilia candidate is progressing. The Philadelphia-based biotech stopped providing updates on its clinical testing since announcing a deal in February to be acquired by Roche. But that $4.8 billion acquisition has been held up for months by the Federal Trade Commission.

A portion of that scrutiny is presumably over Roche acquiring a gene therapy candidate while also marketing Hemlibra, a prophylaxis factor treatment first approved in 2017. Hemlibra has quickly become a key drug for the Swiss pharma, recording $930 million in sales through the first nine months of 2019.

As FTC mulls the deal, Spark has pushed off sharing interim updates on its gene therapy SPK-8001. While company slides presented at the beginning of 2019 showed plans for a midyear update, that never materialized.

"The guidance we provided about a hemophilia A update in mid-2019 was made prior to the announcement of the pending Roche acquisition," a Roche spokesperson wrote to BioPharma Dive in July. "We have no further comment at this time while we focus on completing the transaction."

In the months since, the biotech has stayed quiet. The latest clinical update is more than a year old, with a data cut-off of Nov. 2, 2018 for its Phase 1/2 trial. Those results included 12 patients, with seven tested at the high dose.

While most patients stopped suffering from bleeds and needing infusions, one patient treated with SPK-8001 was hospitalized and needed adrenocortical steroids. Spark stated it planned to include prophylactic steroids going forward in testing.

Spark has started a run-in study for the Phase 3 program, the company disclosed in its first quarter financial filing. While the company has not clarified where that program now stands, a federal database of clinical trials shows the lead-in study actively recruiting for 55 patients with a completion date of July 2020.

Sangamo has been the wild card in the race with impressive, albeit early, efficacy and safety data.

Early results from a Phase 1/2 trial turned heads in April by showing a small handful of patients treated with SB-525 had normal Factor VIII activity levels six weeks after treatment. And a further update in July backed that result up, showing more patients sustaining normal levels of the essential blood-clotting protein, so far at least.

Pfizer will take over late-stage testing of SB-525 through a 2017 licensing deal with Sangamo. CEO Albert Bourla said it has completed transferring the manufacturing process and enrolled the first patient in October for a six-month lead-in trial for the Phase 3 program.

Phase 3 dosing is expected to begin in the first half of 2020, Bourla said on Pfizer's third quarter earnings call.

For its part, Sangamo will also present updated data from its Phase 1/2 trial on Dec. 7 at the American Society of Hematology's annual meeting in Orlando, Florida.

The hemophilia gene therapy race is poised to evolve in 2020. BioMarin could submit valrox for potential approval, while industry leaders in Roche and Pfizer will both be working to advance Spark's and Sangamo's products, respectively.

And while BioMarin has a sizable lead, the biotech will still need to capitalize on its pole position to gain FDA approval and establish the market.

Go here to see the original:
The hemophilia gene therapy race faces a critical year in 2020 - BioPharma Dive