Twelve Indicted in Kickback Conspiracy, Former CEO Pleads Guilty

TEXARKANA, Texas Twelve individuals from three states have been charged in a federal indictment returned in the Eastern District of Texas, announced U.S. Attorney Joseph D. Brown today.

Nicolas Arroyo, 38, of Newport Coast, CA, pleaded guilty to conspiracy to defraud the United States on Jan. 14, 2020 before U.S. Magistrate Judge Caroline Craven.

According to information presented in court, Arroyo was the CEO of a clinical laboratory when he conspired with others to pay and receive kickbacks in exchange for the referral of and arranging for health care business, specifically pharmacogenetic (PGx) tests. Pharmacogenetic testing, also known as pharmacogenomic testing, is a type of genetic testing that identifies genetic variations that effect how an individual patient metabolizes certain drugs. The illegal arrangement concerned the referral of PGx tests to clinical laboratories in Fountain Valley, California, Irvine, California, and San Diego, California. More than $28 million in illegal kickback payments were exchanged by the defendants and others during the conspiracy. On Dec. 11, 2019, a federal grand jury returned an indictment in which Philip Lamb, 44, of Scottsdale, Arizona; Nicolas Arroyo, 38, of Newport Coast, California; Vincent Marchetti, Jr., 55, of Coronado, California; William Flowers, 55, of Houston, Texas; Steven Donofrio, 45, of Temecula, California; James J. Walker, Jr. a/k/a Jimmy Walker, 46, of Frisco, Texas; Timothy Armstrong, 62, of Frisco, Texas; Virginia Blake Herrin, 54, of Frisco, Texas; Patrick Ridgeway, 50, of Jackson, Mississippi; Chismere Mallard, 39, of McAllen, Texas; Ray W. Ng, 61, of Dallas, Texas; and Ashley Kretzschmar, 34, of Aledo, Texas; were indicted for conspiracy to commit illegal remunerations in violation of the Anti-Kickback Statute. The Anti-Kickback Statute prohibits offering, paying, soliciting, or receiving remunerations in exchange for the referral of or arranging for items or services payable under federal health care programs.

We continue to see individuals in the healthcare industry creating illegal kickback arrangements, trying to cheat the system and turn healthcare decisions into financial decisions instead of what is best for the patient, said United States Attorney Joseph D. Brown.This must stop, and doctors should be aware of the emphasis that is being put on stopping these practices.

Under federal statutes, Arroyo faces up to 5 years in federal prison at sentencing. The maximum statutory sentence prescribed by Congress is provided here for information purposes, as the sentencing will be determined by the court based on the advisory sentencing guidelines and other statutory factors. A sentencing hearing will be scheduled after the completion of a presentence investigation by the U.S. Probation Office.

This case was investigated by the U.S. Department of Health and Human Services, Office of Inspector General, the FBI Dallas Frisco Resident Agency, and the U.S. Department of Homeland Security, Homeland Security Investigations. It was prosecuted by Assistant U.S. Attorneys Nathaniel C. Kummerfeld and L. Frank Coan, Jr.

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Twelve Indicted in Kickback Conspiracy Former CEO Pleads Guilty - Twelve Indicted in Kickback Conspiracy Former CEO Pleads Guilty TEXARKANA Texas ...

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If you're feeling down and uninspired right now, you're not alone. The winter blues -- including its most severe form, Seasonal Affective Disorder, or SAD -- really is a thing.

According to Rush University Medical Center, about 14% of Americans get the winter blues, while 6% wrestle with full-blown SAD. The good news is, these are treatable conditions, once you know the basics.

Many of us experience mood shifts during the long, dark days of winter, but if you're experiencing the following symptoms over an extended period, it may be something more:

You feel sluggish and can't get going.

You've lost interest in activities you usually enjoy.

You prefer to hibernate instead of socialize.

You're overeating (carbs cravings, anyone?), or conversely, have lost interest in food.

You're not sleeping well.

You feel hopeless or worthless.

Where's the line between simply the winter blues and an all-out case of SAD? Often, it's a matter of degree. With the winter blues, you can still function and go about your day. However, you may have SAD if:

You're unremittingly depressed or anxious.

You're having problems getting through your day.

You're abusing alcohol or other substances.

You're having suicidal thoughts.

In either case -- but especially if symptoms are serious -- seek professional help, starting with your primary care physician.

These are truly seasonal disorders. Science reveals that a lack of exposure to sunlight -- in short supply during fall and winter -- can wreak havoc on our biological clocks.

How it works: Sunlight triggers our brains to release serotonin, a powerful hormone that regulates mood. Less sunlight results in less serotonin and melatonin, which is key to a good night's sleep.

The further you live from the equator, the greater the risk. For those of us in the Chicago area, where days are short but winter is long, the risk is real.

Luckily, there are several successful treatment options, including:

Light therapy -- During light therapy, you sit in front of a special light box that mimics natural daylight. You can buy a light box without a prescription, but talk to your doctor first to learn what features to look for and how to use it.

Exercise -- Research shows exercise increases serotonin and endorphin production. Many doctors recommend exercising at least 30 minutes on most days.

Cognitive Behavior Therapy -- This effective form of psychotherapy helps people replace negative thoughts and behaviors with healthier habits.

Medication -- Some doctors prescribe antidepressants to regulate chemical imbalances caused by SAD. Never self-medicate; always consult your doctor.

If you don't have the winter blues, give thanks -- and practice good prevention by soaking up as much daylight as you can. When your schedule permits it, start the day with a crisp morning walk -- or at least get outside and moving at lunchtime. Grab a friend, or your dog, or your friend's dog.

And if you think you need help, ask for it. Don't wait for this to go away -- it's a way off until spring.

Teri Dreher is board-certified patient advocate. A critical care nurse for more than 30 years, she recently founded Seniors Alone Guardianship & Advocacy Services (SeniorsAlone.org), a nonprofit organization that serves the area's senior orphans. She also is the founder of NShore Patient Advocates, http://www.northshorern.com.

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Don't underestimate the winter blues - Chicago Daily Herald

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While popping a pill may sound promising to us sleep-deprived, walking zombies, there is little evidence to show that sleeping pills actually add very much shut eye to your daily count, not to mention the dangerous side effects that can occur from misuse.

That being said, there are some sleep trackers, sleep apps and natural sleep aids that can make dozing off and staying asleep a little bit easier. Here are some of our top picks:

"In order to create a conducive sleep environment we want it as dark as possible and the blackout curtains will block out warming light for people who are sleeping when the sun is shining, or in the summer when it starts to get light earlier. Street lights and city lights can also interfere with sleep," says Dianne Augelli, MD, a sleep physician at Weill Cornell Medicine in New York. "We generally want our rooms to be dark, quiet and cool. The idea is to use the blackout curtains so that your sleep isnt disturbed prior to your wake time. So if youre someone who is getting up at 7:30 a.m., but its already light at six, that extra light may cause awakening that can fragment your sleep."

If you live in an urban location, or have some loud neighbors, that's even more incentive to consider a new set of curtains. The Eclipse curtains arent only a decorative accent in the bedroom, they block over 99 percent of intrusive light and reduce unwanted noise making your sleep more restful.

Its not just the light coming from our devices, but other lights in the room, that can keep you awake.

I advise turning down the lights in your home towards the end of the day," says Dr. Apostolos Lekkos, found of Bios Functional Medicine. "Lower light levels can help promote your natural melatonin production your sleep hormone.

C-Sleep is a blue tooth enabled light bulb that supports your body's natural sleep cycle by emitting a calm light at night and vibrant light in the morning. You can instantly dim or brighten the bulb, without even having to get out of bed, thanks to a handy app. Plus, you can create schedules and groups to control multiple bulbs at the same time.

We've all been there: We're awakened from our sleep by the heat, and rustle around kicking off our socks and tossing the comforter to the floor. You curse your partner who insists on keeping the air off because they're always cold. What if we told you there is a sheet for that?

The Temperature Regulating Sheet Set is the ultimate solution for thermally incompatible couples. It uses the Outlast fabric technology, which was developed for NASA to deal with temperature fluctuations in space. The "phase change" fabric absorbs and stores excess heat from your body. When you cool off, it releases the heat back to your body to maintain a consistent temperature all night long.

"We definitely want you to be cool when you're sleeping. If you're too hot and you feel that the particular sheets that you have are making you too hot, then you can consider a different type of sheet," says Dr. Augelli. "Some people will swear by [temperature regulating sheets], others will notice no change. But people need a more breathable sheet. The National Sleep Foundation recommends 200-400 thread count sheets, which allow some air movement."

This set from Sharper Image clocks in at 300 thread count so if you find yourself waking up sweating at night, they are worth a try.

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Philips SmartSleep, which connects to your home Wi-Fi, helps improve your sleep quality and helps you learn how the environment of your bedroom affects your rest. You'll be interacting with its proprietary app SleepMapper to document and receive feedback on some of your behaviors.

The best cure for being unable to fall asleep is utilizing a sleep tracker which is available through many apps," says Lindsey Huttner, LCSW, psychotherapist, located in Queens, NY. "In the app you will document when you get into bed, approximate the time you fall asleep, how long you sleep, and how many times you woke and for how long. The app will then 'prescribe' how much sleep you should aim to get."

In addition to tracking your sleep patterns, SmartSleep works with its companion app to track and monitor your bedroom's temperature, noise, light and humidity levels. The light it emits is designed to ease you out of sleep in a healthy and productive way.

While Augelli is hesitant to recommend trackers that record sleep time (and unrest) due to a lack of evidence and accuracy, she does say that tracking your sleep patterns can be helpful for some.

"They will give you some idea of your sleep, but they arent really able to tell the difference between [REM sleep and deep sleep] with the technology thats available on them," she says. "It can be helpful if youre tracking your number of hours of sleep and making sure you get adequate sleep and that youre going to sleep around the same time. That can be a problem with a lot of people, just not sleeping enough hours. If youre not getting your seven hours of sleep then this can help track for that."

The Versa 2 is optimized with a sleep mode to mute troublesome notifications. It also provides you with a Sleep Score, a simple way to see how you slept the night prior based on the metrics it's detecting, from sleep time to wakefulness and duration of sleep. Essentially, you'll gamify your sleeping habits to improve them.

Your sky-high stress levels may be to blame for all the tossing and turning, but that lumpy pillow sure isn't helping the cause. Before you run to Bed Bath and Beyond and grab a new one, consider investing in a pillow that works double duty. The Eden Pillow is designed to provide you with better quality sleep. And the gel-infused memory foam and microfiber fill are blended to offer cooling properties.

If you just can't seem to part with your nightly Netflix ritual, you should wear blue light blocking sunglasses while watching television, advises Dr. Lekkos.

"There are some small studies that show that the time it takes to fall asleep has been reduced for people who wore the light blocking glasses for several hours before bedtime," adds Augelli. "What you want to do ideally, is put away all your work and phone an hour before bed, in the process of your wind down routine. But if you have to work, you want to employ whatever you can to reduce the amount of impact it will have. Blue light blocking glasses reduce exposure to blue light that suppresses melatonin, which is our natural hormone that helps with sleep; the decrease in melatonin can prevent you from falling asleep."

The glasses filter out the high energy visible (HEV) blue-violet light from backlit screens, helping to ease the disruption is has on your sleep cycle. Keep a pair next to your bed, ready to grab and slip on when you get sucked into that late-night "Friends" marathon.

"For some people sound is soothing and relaxing and helps deactivate them for sleep," says Augelli. "Some of them will act to cover up other environmental noise, so in those cases it can be helpful, you just want to make sure there is no big change in frequency of that sound. (For example, on the TV when theres a commercial thats much louder.) We generally recommend white noise machines because they are pretty stable, but it is helpful for some people to deactivate if they hear something very soothing and thats part of their wind down routine and they feel comfortable with that."

The Marpac Dohm's natural white noise is emitted by its built-in fan, rather than relying on digital recordings. You can choose between two speed options and adjust its tone and volume to reach the perfect level of white noise for you.

According to Michael Gelb, DDS, MS, co-author of the new book "GASP!: Airway Health - The Hidden Path to Wellness," more than half of us struggle with breathing through our noses. This struggle often results in snoring. And even if you're part of the lucky half who has no problem breathing at night, there's a good chance your partner's issues are affecting your quality of sleep. Time to trade in the shaking and kicking for tossing a Mute Snoring device their way.

By holding open the nasal airways like a stent, Mute helps noisy snorers and poor nasal breathers by increasing airflow in the nose. You can individually adjust the device for each nostril, so it's comfortable while you sleep. And user trials are promising: 75 percent of people reported less or much less snoring while using Mute.

If youre prone to checking email, or watching Netflix, on your laptop in bed at night, you may want to have your computer screen follow suit with reducing blue light. You can run apps like f.lux on your laptop or computer that will automatically change the screen color as the day goes on, says Lekkos.

Like Night Shift, f.lux uses your location to synchronize your computer with the rising and setting of the sun. At night, f.lux slowly adjusts the colors on your computer screen to a warmer hue that's supposed to be less abrasive on the eyes (especially if you're in an otherwise dark room) and helps reduce your exposure to that blue light that keeps you awake.

"Any platform that will reduce the amount of blue light that is emitted from your electronic devices will be helpful," says Augelli. "I definitely recommend employing them."

Smartphone apps aren't adequate substitutes for assistance from a professional but they can be helpful adjuncts to treatment for those who wish to track sleep time and quality or for promoting greater physical relaxation before bed, says Joel Minden, Ph.D,, clinical psychologist at the Chico Center for Cognitive Behavior Therapy. "CBT-i Coach is nice because it includes a sleep diary. One of the most effective treatments for insomnia is sleep restriction therapy, and tracking sleep data is an important part of the process.

The app was designed for those who have experienced symptoms of insomnia and are looking to improve their sleep habits. It guides users through the process of learning about sleep, developing a better sleep routine and improving sleep environments. Plus, it has a structured program that teaches strategies proven to help alleviate symptoms of insomnia.

In fact, one study found that 60 percent of doctors who used the app in conjunction with treatment for insomnia found it favorable in encouraging people to adhere to instructed techniques.

While you're staring at the ceiling at 1 a.m., ever think back to those wonderful days when your mom would read you a story and you'd be out cold two pages in? Well, maybe a little bedtime story is in order. Enter: Calm, a top-rated meditation app that recently launched a new feature to the platform called "Sleep Stories," which are essentially bedtime tales for adults.

You may recognize some of your storytellers: Matthew McConaughey narrates one about the mysteries of the universe, childhood favorite LeVar Burton take us on a journey across the solar system, and Steven Fry (the narrator of all 7 "Harry Potter" books) takes us through the lavender fields of France.

Way too many people are on their smartphone, tablet, reading device or watch TV just before going to bed. What people do not realize is that there is a blue light that is emitted from these devices that (when it hits the back of the eye) will stimulate brain waves that promote our 'awake' state of mind, thus making it very difficult to fall and stay asleep, says Lekkos. Many smartphones now have the 'Night Shift' feature that you can turn on to block this blue light.

To enable the feature on your iPhone, go to Settings, Display & Brightness, and then schedule Night Shift for the hours you will be winding down for bed (and sleeping). By doing this, you're telling your phone to move the color spectrum from cooler (blue) towards warmer (yellow) to limit your exposure to sleep-disrupting blue light at night. It will then readjust the tones in the morning when the sun rises.

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Dublin, Jan. 14, 2020 (GLOBE NEWSWIRE) -- The "Japanese Biosimilars Landscape Study, 2009-2020" report has been added to ResearchAndMarkets.com's offering.

In this report, we attempt to analyze trend/requirement of regulatory approval of biosimilars based on Ex-Japan clinical trialdata, factors responsible for each key launched biosimilar penetration (Made in Japan vs. Tested by Japan), and the future competitive landscape in the biosimilars space in Japan.

We also attempt to analyze detail BS market of EPO, Filgrastim/Pegfilgrastim/Insulin/Lantus apart from other key complex Mab Biosimilar opportunities and list out niche opportunities in biosimilar space in Japan. Details of all major consolidation activities done by JP/Foreign companies in biosimilar space in the last five years and crisp summary on strategies of each key player (~21 JP local companies, multinational companies), their interest & focus for future collaboration in biosimilar space.

Since 2009, 25 biosimilars of 12 originator products are approved in Japan and have yielded mixed performances and attained annual sales of ~32.4b ($300m).

Unique biosimilars landscape with the entry of NESP biosame by originator Kyowa Hakko Kirin through its subsidiary, co-promotion/marketing collaboration with local companies for the front-end skill sets (Ayumi, Kyowa Hakko Kirin, Teijin) and few skipped listing to better manage constant supply (Pfizer), a key requirement by MHLW, makes Japan a distinct market vs. US and EU.

Less stringent regulatory environment vs. US for approval, increasing healthcare burden and strong foothold of the marketers have played key roles in this early uptake which is at par to one of the best generic small molecule penetration in Japan in a short time (Filgrastim BS-volume share~45% in two years, Lantus BS- ~9% in 2 months vs. Lipitor generics ~50% volume share).

While analyzing the launched biosimilar penetration since 2009 in Japan, bolstering uptake of Enbrel, Rituxan, and Lantus biosimilar vs. very slow uptake of Remicade BS mainly been attributed with the use of biosimilars in DPC hospitals,product reimbursement under high cost medical care benefit system and front end presence of the local partner.

Despite the string of recent biosimilars approvals, healthcare professionals still harbor concerns over the quality of biosimilars.

MHLW's announcement to update a decade back biosimilars guideline by FY2020 to reduce cost of development as well as to increase confidence of physician on quality, is indicative of biosimilar as important weapon for Chuikyo(Central social Insurance Medical Council) to curb healthcare cost. Further, Biosame pricing game will play a major role in the future for biosimilar penetration. We see that Abenomics measures and government involvement in biosimilars use would lead to the Biosimilars promotions in the coming times in Japan.

Mixed strategies by originator for Bio-same launch (Kyowa Hakko Kirin to launch Biosame vs. Chugai said not to launch Biosame), therapy area wise biosimilars cherry picking by mid-size Japanese companies, and different strategies by local generic companies (NichiIko heading for global market vs. Sawai testing through co-promotion and Towa yet not decided to enter), demonstrates each company's different need and approach to cater biosimilar opportunities in Japan.

Since the last five years, most of the companies have some alliance in place for biosimilars, with most of the Japanese companies undertaking pacts with South Korean companies to ride on their back of biosimilar mAb expertise.

There is a trend of doing product specific alliance by most of the JP companies active in BS space and to go step by step on this high risk/high return opportunities. Against this backdrop, multinational companies like Pfizer are setting their sights on this market without local partnership taking advantage of pro-biosimilar environment to capture decent biosimilar market share. Overall, in the Japan market, each opportunity has a different competitive landscape for itself, and some companies are looking for niche opportunities in biosimilar space as per their specialty therapy area- like ophthalmology BS (Lucentis), Enzyme therapy BS (JCR).

While launched biosimilars now generates ~32b ($300m) sales and its penetration is accelerating, MHLW's approval of Biosame of NESP based on same clinical data as the originator NESP, and current ongoing dialogs to price Biosame higher than biosimilars, indicative of Biosame to be the key hurdle in the future for mid-size biosimilar companies in Japan. In the year 2020, MHLW's stand on Biosame, will be important to decide future of theses mid-size /generic biosimilar developers. Around ~550b opportunity is opening for biosimilar in the next 7 years in Japan due to patent expiry of Wave 2-3 biologics.

Key Topics Covered

CHAPTER 1: EXECUTIVE SUMMARY

CHAPTER 2: JAPANESE GOVERNMENT AND CHUIKYO INITIATIVES TO INCREASE BIOSIMILAR PENETRATION IN JAPAN

CHAPTER 3: LESSONS FROM LAUNCHED BIOGENERIC PROGRESS IN JAPAN SINCE 2009

CHAPTER 4: GROWTH HORMONE MARKET (SOMATROPIN) IN JAPAN

CHAPTER 5: ANEMIA MARKET IN JAPAN

CHAPTER 6: FILGRASTIM (GRAN) BS

CHAPTER 7: REMICADE (INFLIXIMAB) BS

CHAPTER 8: LANTUS (INSULIN GLARGINE) BIOSIMILAR

CHAPTER 9: ENBREL (ETANERCEPT) BIOSIMILAR

CHAPTER 10: ONCOLOGY BIOSMILARS ERA STARTS WITH RITUXAN (RITUXIMAB) BIOSIMILAR

CHAPTER 11: HERCEPTIN (TRASTUZUMAB) BIOSIMILAR

CHAPTER 12: AVASTIN (BEVACIZUMAB) BIOSIMILAR

CHAPTER 13: FABRAZYME (AGALSIDASE BETA) BIOSIMILAR

CHAPTER 14: FORTEO (TERIPARATIDE) BIOSIMILAR

CHAPTER 15: NEXT WAVEOF BIOSIMILAR OPPORTUNITIES IN JAPAN

CHAPTER 16: BIOSIMILAR/BIOPHARMA CMO OPPORTUNITY

CHAPTER 17: REGULATORY APPROVAL REQUIREMENT FOR COMPLEX MAB BIOSIMILAR IN JAPAN

CHAPTER 18: LICENSING/CONSOLIDATION ACTIVITIES IN BIOSIMILAR SPACE IN JAPAN

CHAPTER 19: JAPANESE COMPANIES ACTIVE INTO BIOGENERICS SPACE

For more information about this report visit https://www.researchandmarkets.com/r/edigvv

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Analysis on Japan's Biosimilars Landscape, 2020 - In the Next 7 Years, Biosimilars Will Pose a ~$550B Opportunity in Japan, Due to the Patent Expiry...

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RIYADH: Professor Selwa Al-Hazzaa is a Saudi female success story set on the road to excellence from childhood.

Speaking to Arab News, Al-Hazzaa, an ophthalmologist and chairman of the ophthalmology department at King Faisal Specialist Hospital and Research Center (KFSHRC), told of her 27 years of devoted work to bettering the health care system, becoming the first woman to hold a high position at the hospital where she dedicated her life, energy and time to making a difference in her field.Al-Hazzaas career took off in 1995, as the first Saudi woman to be made a member of the Medical Advisory Council at King Faisal Hospital. Her journey wasnt the easiest, but with her talent, hard work and ambition, she was recognized by the Saudi leadership early on and used the platform to pave the way for future women in medicine and other fields.Born into a family of five girls, she grew up in Tucson, Arizona in the 1960s while her father was completing his studies. She excelled in her school years, always living up to the highest standards and expectations which she has placed upon herself.

I didnt choose ophthalmology, ophthalmology chose me.

Prof. Selwa Al-Hazzaa

I went into medicine not wanting (to do) it, she said. Nevertheless, she put all her energy into studying, because she had a higher ambition and was keen to make a difference.One of her biggest challenges was when it was time for her to enroll in university. She wanted to travel abroad to study, but was unable to, because it was rare for women to do so at the time.Back then the only two real professional options women had were medicine or education, and her father gave her a choice: Either to become a teacher or a physician. She chose the latter.After obtaining her medical degree from King Saud University, she did her fellowship at the Wilmer Ophthalmologic Institute at Johns Hopkins University School of Medicine, in Washington, DC.

HIGHLIGHTS

Selwa Al-Hazzaa became the first woman to hold a high position at the hospital where she dedicated her life, energy and time to making a difference in her field.

With her talent, hard work and ambition, she was recognized by the Saudi leadership early on and used the platform to pave the way for future women in medicine and other fields.

Her first patient was a 9-year-old Saudi girl born blind, a case Al-Hazzaa had followed since the girl was less than a year old.

She returned to the Kingdom, where she was later chosen by the head of KFSHRC, Dr. Anwar Jabarti, to be the late King Fahds ophthalmologist. She credits Jabarti for realizing her potential, dedication and skills by looking beyond gender and solely at talent.Her dream of representing her country came true, though under sombre circumstances, when she went on her first diplomatic mission after the fatal Sept. 11 2001 terror attacks in the US, remembering her fathers words: When people trust you, they will then let you represent the country.

Selwa Al-Hazzaa. (AN photo by Ali Aldhahri)

And represent her country she did, as she was the only woman between men, and with no training whatsoever in the political arena, she spoke from the heart, connecting with people. From that day on, the government took me as their voice of Saudi Arabia after Sept. 11.Through a lifetime of giving, people would ask her what was the secret to her success. There is no secret females are always givers. When we are young, we take care of our siblings, when we are married we take care of our husbands, we get pregnant and take care of our children, she said.Elected as an executive member of the International Council of Ophthalmology (ICO) in 2002, she became the youngest member, the first woman member from the Middle East, and the only female on the council from 2002-2006. She stood down in 2010.

NUMBER

2.2bn - There are an estimated 2.2 billion people with vision impairment or blindness globally, with an estimated 1 billion who suffer from moderate or severe distance vision impairment or blindness (WHO 2019).

In 2017, Al-Hazzaa was granted the degree of doctor of humanities, honoris causa, the highest honor at Franklin University, one of many honorary titles shes received in her career. She is also a member of various editorial boards, fellowships and committees, and was one of the first group of women appointed to the Saudi Shoura Council by late King Abdullah bin Abdul Aziz in 2003, in a historic move, allowing women for the first time to be part of the Kingdoms formal advisory body.

FASTFACT

Last November, Selwa Al-Hazzaa, alongside her colleague Dr. Mohamed Khuthaila and a medical team consisting of entirely of Saudis, put the Kingdom on the map as the first country in the Middle East, and the 5th globally, to utilize LUXTURNA, the first USA FDA-approved gene therapy treatment for any genetic disorder to treat blindness in children.

With her 27 years of experience in the field, publishing 69 accredited papers and more, her lifes work finally paid off in November of last year when she, alongside her colleague Dr. Mohamed Khuthaila and a medical team consisting entirely of Saudis, put the Kingdom on the map as the first country in the Middle East, and the 5th globally, to utilize LUXTURNA, the first USA FDA-approved gene therapy treatment for any genetic disorder to treat blindness in children.Her first patient was a nine-year-old Saudi girl born blind, a case Al-Hazzaa had followed since the girl was less than a year old. The successful utilization of the treatment was one of her finest career achievements to date.If you are going to take a certain specialty, dont take what everybodys taking take something that doesnt exist and make it exist. Take something hard, because then when you are called upon, it will be regardless of your gender.I didnt choose ophthalmology, ophthalmology chose me.

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Selwa Al-Hazzaa: The Saudi doctor giving the gift of sight - Arabnews

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"We're moving close to a cure," says Wyatt's doctor, Mark Tarnopolsky, who is director of the neuromuscular and neurometabolic clinic at McMaster University Medical Centre in Hamilton.

Children with spinal muscular atrophy lack a certain protein that is critical for the maintenance and function of specialized nerve cells, called motor neurons, which control muscle movement throughout the body.

Without the protein, the motor neurons die and the muscles can't move. If they don't move, they shrink and weaken. The child suffers debilitating effects that inhibit even their ability to breathe and swallow.

Until recently, most children with this type of spinal muscular atrophy died of respiratory failure by their second birthday.

But in 2018, a new drug called Spinraza was approved for use in Canada. It prevents the rapid nerve degeneration from occurring, if it is given early enough in the child's life.

At $125,000 a dose, it's very expensive. Luckily, the Ontario government pays for it.

Wyatt has had four doses since he was diagnosed six months ago.

He can clap his hands together and kick a little bit, Dannon said.

"He is such a happy little guy," she said. "Every moment I spend with him, I love."

Wyatt's aunt and parents have started a GoFundMe page to help pay for some of his expenses. The family has already raised about $74,000 through this and other fundraisers.

They've poured their hopes into a brand new drug called Zolgensma. It's a gene therapy does the same thing as Spinraza, but it's a single dose that lasts 25 years.

"With a longing we didn't know was possible, we hope one day Wyatt can dance to the beat of his favourite song, send our hearts racing as he climbs a high tree or wrestle with his dad on the floor," the family says on its GoFundMe page.

But that's unlikely to happen.

The drug is very expensive, at $2.8 million Canadian for a dose. It's not available in Canada yet.

More significantly, Tarnopolsky thinks the benefits would be very limited for a child like Wyatt. That's because he's already too old.

Nerve degeneration happens very quickly for the tiny number of children, perhaps several hundred in Ontario, who have this disease. It drops "like a stone" shortly after birth, Tarnolpolsky said.

And once the nerve function is gone, it doesn't come back.

The studies have shown it's much better for children to receive either Spinraza or Zolgensma if they get it when they're two or three months old. The oldest child in one of the Zolgensma studies was eight months old.

He would like to see newborns routinely screened for this disease so it can be treated right away.

Michael Harris, the Waterloo regional councillor who was formerly MPP for Kitchener-Conestoga riding, has met many families like the Vaseys.

"They'll do anything" for a chance for their child to get better," he said.

When he was MPP between 2011 and 2018, Harris championed the cause of families with rare diseases.

He toured the province to hear from them, tried to have an all-party committee of MPPs to discuss the issues, and lobbied for them to have access to government funds for the expensive medication they need. (It's expensive because so few patients need it, and there are few buyers to share the high cost of research, development and clinical trials.)

Working for these families was "the only time I came to tears in my entire career," he said. "I think of them all the time."

One look at Wyatt helps explain why.

He is cheerful all the time despite the long uphill road ahead for him, his family said.

Now that he has learned to clap his hands together, "all he loves to do now is clap," said Dannon.

"You would never know any of the challenges (that he faces) exist for him."

ldamato@therecord.com

Twitter: @DamatoRecord

ldamato@therecord.com

Twitter: @DamatoRecord

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A baby who can't crawl: A cure is on the way, but this Milton baby's time is running out - InsideHalton.com

Recommendation and review posted by Bethany Smith

The "North America Hormone Replacement Therapy Market: Industry Trends, Share, Size, Growth, Opportunity and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The publisher expects the market to reach a value of US$ 4.7 Billion by 2024, registering a CAGR of 5.35% during 2019-2024.

The North America hormone replacement therapy market reached a value of US$ 3.5 Billion in 2018. Hormone replacement therapy (HRT) is used for replenishing hormones that are present in low levels in the human body. This treatment is particularly favorable for patients who are experiencing growth hormone deficiency, women nearing menopause and older people suffering from hypogonadism. HRT is available in several forms such as gels, injections, implants, and skin and mouth patches (transdermal). However, it may not be suitable for patients that have a record of blood clots, liver disease and untreated high blood pressure.

North America hormone replacement therapy market is currently being driven by several factors. A surge in the incidences of hormone imbalance disorders, especially in the geriatric and neonatal populations, is spurring the demand for HRT in North America. In line with this, the rising need for new treatment options with better safety results is further catalyzing the market growth in the region. Apart from this, increasing R&D activities for hormone replacement products is enhancing their quality and efficiency. Additionally, the increasing consumer awareness, coupled with the rising technological innovations, such as new gel-based formulations, have also spurred the demand for hormone replacement products in the region.

Key Questions Answered in This Report:

Report Coverage:

Key Topics Covered:

1 Preface

2 Scope and Methodology

2.1 Objectives of the Study

2.2 Stakeholders

2.3 Data Sources

2.3.1 Primary Sources

2.3.2 Secondary Sources

2.4 Market Estimation

2.4.1 Bottom-Up Approach

2.4.2 Top-Down Approach

2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

4.1 Overview

4.2 Key Industry Trends

5 Global Hormone Replacement Therapy Market

5.1 Market Performance

5.2 Market Breakup by Product

5.3 Market Breakup by Route of Administration

5.4 Market Breakup by Type of Disease

5.5 Market Breakup by Region

5.6 Market Forecast

6 North America Hormone Replacement Therapy Market

6.1 Market Performance

6.2 Market Forecast

7 North America Hormone Replacement Therapy Market: Breakup by Product

7.1 Estrogen Replacement Therapy

7.2 Human Growth Hormone Replacement Therapy

7.3 Thyroid Replacement Therapy

7.4 Testosterone Replacement Therapy

7.5 Others

8 North America Hormone Replacement Therapy Market: Breakup by Route of Administration

8.1 Oral

8.2 Parenteral

8.3 Transdermal

9 North America Hormone Replacement Therapy Market: Breakup by Type of Disease

9.1 Menopause

9.2 Hypothyroidism

9.3 Male Hypogonadism

9.4 Growth Hormone Deficiency

9.5 Others

10 North America Hormone Replacement Therapy Market: Breakup by Country

10.1 United States

10.1.1 Historical market Trends

10.1.2 Market Breakup by Product

10.1.3 Market Breakup by Route of Administration

10.1.4 Market Breakup by Type of Disease

10.1.5 Market Forecast

10.2 Canada

10.3 Mexico

11 SWOT Analysis

11.1 Overview

11.2 Strengths

11.3 Weaknesses

11.4 Opportunities

11.5 Threats

12 Value Chain Analysis

12.1 Overview

12.2 Research and Development

12.3 Raw Material Procurement

12.4 Manufacturing

12.5 Marketing

12.6 Distribution

12.7 End-Use

13 Porter's Five Forces Analysis

13.1 Overview

13.2 Bargaining Power of Buyers

13.3 Bargaining Power of Suppliers

13.4 Degree of Rivalry

13.5 Threat of New Entrants

13.6 Threat of Substitutes

14 Competitive Landscape

14.1 Market Structure

14.2 Key Players

14.3 Profiles of Key Players

For more information about this report visit https://www.researchandmarkets.com/r/xr45u2

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005380/en/

Contacts

ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.com For E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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North America Hormone Replacement Therapy Market Expected to Generate a Value of US $4.7 Billion by 2024 - ResearchAndMarkets.com - Yahoo Finance

Recommendation and review posted by Bethany Smith

Also, I think we're all trying to be kinder to the environment with our lifestyle choices, and unlike most sheet masks on the market, it's made with100 per cent natural cotton fibres that will biodegrade six months after use. The packaging itself is recyclable too.

If you've ever watched one of those videos of a baby smiling with glee after they try chocolate for the first time, that was me with chemical exfoliation. Unlike scrubs which use psychical exfoliation to buff away at dead skin, chemical exfoliants like AHAs, glycolic acid and lactic acid dissolve dead skin and sebum which can cause breakouts and dull skin.

And when it comes to products which do just that, this cult-favouritemask is one of my personal picks.

Not only does it kind of smell, and look, like theinside of a pumpkin pie, the wonderful combo of glycolic acid, fruit stem cells, Vitamin C, Manuka honey andantioxidant-rich pumpkin puree works to give you the deliciously radiant skin you crave.

In terms of tingliness, there was less sensation than the CannaCell Glow Mask but the results were equally as impressive. My pores appeared tighter, and the texture of my skin felt smoother, brighter and just... better.

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"I tried four natural face masks in one week. Here's exactly what they did for my skin." - Mamamia

Recommendation and review posted by Bethany Smith

Experts from the Mayo clinic warned that spots on the skin may signal a blood cancer. In connection with what experts suggest as soon as possible to go to the doctor when the symptom of.

Leukemia is a cancer primarily occurs in the bone marrow as a result of mutations in blood stem cell. The consequence is the loss of the descendants of the mutated cells ability to differentiate to Mature blood cells. The danger of the disease is that the symptoms are not specific, often among the signs unexplained weight loss, fever and chills. The experts considered it important to warn you that spots on the skin can indicate cancer of the blood that allows an early identification is a deadly health hazard. Among the other important symptoms of blood cancer: swollen lymph nodes, enlarged liver or spleen, frequent nosebleeds, excessive sweating, especially at night, bone pain, constant fatigue, recurrent infections.

With regard to treatment, the experts from Mayo clinic said: chemotherapy is the main form of treatment. Biological therapy works by using methods that help the immune system to recognize cancer cells and attack them. Among the methods of struggle with a deadly disease and radiation therapy, which destroys leukemia cells and stop their growth. Among the solutions and stem cell transplantation is bone marrow transplantation. The essence of the procedure is that the blood stream is filled with healthy blood cells, which often helps to restore normal functioning of the hematopoietic system.

Natasha Kumar is a general assignment reporter at the Times Hub. She has covered sports, entertainment and many other beats in her journalism career, and has lived in Manhattan for more than 8 years. Natasha has appeared periodically on national television shows and has been published in (among others) Hindustan Times.? Times of India

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Experts from the Mayo clinic: Spots on the skin can indicate cancer of the blood - The Times Hub

Recommendation and review posted by Bethany Smith

This Cosmetic Skin Care market report endows with a far-reaching survey of key players in the market which is based on a range of objectives of an organization such as profiling, the product outline, the quantity of production, required raw material, and the financial health of the organization. One of the sections in the report covers the evaluation of probabilities of the new investment projects and overall research conclusions are offered. Thus, the transparent, truthful and extensive market information and data included in this global industry report will definitely help develop business and improve return on investment (ROI).

Global cosmetic skin care market is set to witness a substantial CAGR of 5.5% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Increasing self-consciousness among population and rising demand for anti- aging skin care products are the factor for the market growth.

Global Cosmetic Skin Care Market By Product (Anti-Aging Cosmetic Products, Skin Whitening Cosmetic Products, Sensitive Skin Care Products, Anti-Acne Products, Dry Skin Care Products, Warts Removal Products, Infant Skin Care Products, Anti-Scars Solution Products, Mole Removal Products, Multi Utility Products), Application (Flakiness Reduction, Stem Cells Protection against UV, Rehydrate the skins surface, Minimize wrinkles, Increase the viscosity of Aqueous, Others), Gender (Men, Women), Distribution Channel (Online, Departmental Stores and Convenience Stores, Pharmacies, Supermarket, Others), Geography (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Industry Trends and Forecast to 2026 ;

Complete report on Global Cosmetic Skin Care Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Market Definition: Global Cosmetic Skin Care Market

Cosmetic skin care is a variety of products which are used to improve the skins appearance and alleviate skin conditions. It consists different products such as anti- aging cosmetic products, sensitive skin care products, anti- scar solution products, warts removal products, infant skin care products and other. They contain various ingredients which are beneficial for the skin such as phytochemicals, vitamins, essential oils, and other. Their main function is to make the skin healthy and repair the skin damages.

Key Questions Answered in Global Cosmetic Skin Care Market Report:-Our Report offers:-

Top Key Players:

Market Drivers:

Market Restraints:

Key Developments in the Market:

Customize report of Global Cosmetic Skin Care Market as per customers requirement also available.Market Segmentations:Global Cosmetic Skin Care Market is segmented on the basis of

Market Segmentations in Details:By Product

By Application

By Gender

By Distribution Channel

By GeographyNorth America

Europe

Asia-Pacific

South America

Middle East & Africa

Competitive Analysis: Global Cosmetic Skin Care Market

Global cosmetic skin care market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cosmetic skin care market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

About Data Bridge Market Research:Data Bridge Market Researchset forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Contact:Data Bridge Market ResearchTel: +1-888-387-2818Email:[emailprotected]

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Cosmetic Skin Care Market Competitive Insights, Trends and Demand Growth 2019 to 2026 - Food & Beverage Herald

Recommendation and review posted by Bethany Smith

SAN FRANCISCO andPFFFIKON, Switzerlandand LONDON, Jan. 13, 2020 /PRNewswire/ --DiNAQOR AG, a global gene therapy platform company, today announced a research collaboration and exclusive license agreement with UCL to develop novel gene therapies for the treatment of monogenic cardiomyopathies, diseases of the heart muscle that can lead to heart failure in children and adults.

The collaboration will focus on the development of therapies targeting several genes using DiNAQOR's cardiac modular technology platform. Under the terms of the agreement, DiNAQOR and UCL will collaborate to advance DiNAQOR's two discovery cardiac gene therapy programs into clinical development. DiNAQOR will obtain exclusive worldwide rights to any therapies developed as part of the collaboration. If a product is successfully commercialized from the collaboration, UCL will be entitled to royalties on sales.

The license agreement was carried out by UCL's technology commercialisation company, UCL Business Ltd (UCLB), part of UCL Innovation & Enterprise. Based on the research of Professor Thomas Voit at the UCL GOSH Institute of Child Health,the agreement aims to utilise UCL's leading expertise in gene therapy.

"Early stage partnerships are critical to expedite innovation in gene therapy research and development. UCL is one of the world's leading research centers and is ideally suited to help us expand our pipeline of gene therapies for cardiomyopathies," said Dr. Valeria Ricotti, Chief Medical Officer at DiNAQOR.

"This partnership represents an exceptional opportunity to accelerate development of potentially life-saving gene therapies for patients suffering from heart failure. We are excited to collaborate with the DiNAQOR team to help find a cure for monogenic cardiomyopathies," said Dr. Perry Elliott, Professor of Cardiovascular Medicine at The UCL Great Ormond Street Institute of Child Health (GOS ICH).

About Monogenic Cardiomyopathies

Cardiomyopathy is a disease of the heart muscle that can lead to heart failure. Approximately 50% of all cardiomyopathies are caused by a single-gene or monogenic defect. There are 1.7 million people in the European Union and the United States currently affected by a monogenic cardiomyopathy, 300,000 of these individuals have a defect in the MYBPC3 gene. There is currently no cure for patients living with genetic cardiomyopathies.

About DiNAQOR

Founded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001is focused on the treatment of MYBPC3-linkedcardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

About UCL London's Global University

UCL is a diverse community with the freedom to challenge and think differently.

Our community of more than 41,500 students from 150 countries and over 12,500 staff pursues academic excellence, breaks boundaries and makes a positive impact on real world problems.

We are consistently ranked among the top 10 universities in the world and are one of only a handful of institutions rated as having the strongest academic reputation and the broadest research impact.

We have a progressive and integrated approach to our teaching and research championing innovation, creativity and cross-disciplinary working. We teach our students how to think, not what to think, and see them as partners, collaborators and contributors.

For almost 200 years, we are proud to have opened higher education to students from a wide range of backgrounds and to change the way we create and share knowledge.

We were the first in England to welcome women to university education and that courageous attitude and disruptive spirit is still alive today. We are UCL.

http://www.ucl.ac.uk| Follow @uclnews on Twitter | Watch our YouTube channel | Listen to UCL podcasts on SoundCloud | Find out what's on at UCL Minds | #MadeAtUCL

About UCL Business Ltd (UCLB)

UCL Business Ltd (UCLB), part of UCL Innovation and Enterprise, is a leading technology commercialisation company that supports research and innovations arising from UCL, one of the UK's top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL's associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market.

Contacts

DiNAQOR:KWM CommunicationsKellie WalshT: 1-914-315-6072E: kwalsh@kwmcommunications.com

UCL:UCL Media Relations Dr. Rebecca CaygillT: +44 (0)20 3108 3846 / +44 (0)7733 307 596 E: r.caygill@ucl.ac.uk

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SOURCE DiNAQOR

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DiNAQOR AG Announces Research Collaboration and License Agreement with UCL to Investigate Gene Therapies for Heart Failure - P&T Community

Recommendation and review posted by Bethany Smith

Horizon Discovery Group plc (LSE: HZD) ("Horizon", "the Company" or "the Group"), a global leader in the application of gene editing and gene modulation technologies, today announced that it will provide access to a novel base editing technology licensed from Rutgers, The State University of New Jersey, for exclusive use in therapeutic, diagnostic and services applications. This technology is incorporated into Horizons next-generation gene editing platform to enable the development of novel therapeutics that rely on engineering patients cells either directly in the body (gene therapy), or externally before transplanting back into the patient (cell therapy). This platform will also expand the Companys research tools and service provisions.

The Company formed an exclusive partnership with Rutgers in January 2019 to further develop the novel base editing technology invented by Dr. Shengkan Jin, associate professor of pharmacology, and co-inventor Dr. Juan C. Collantes, post-doctoral research fellow at Rutgers Robert Wood Johnson Medical School, and has since been funding research in base editing at the University while undertaking its own evaluation and proof-of-concept studies. Horizon has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking 35 partners to assess and shape the development of its Pin-point base editing platform.

Horizon will offer partners access to a novel system that could be used to progress more effective multi-gene knockout cell therapy programs through clinical development with an improved safety profile. Partners will also gain access to the Companys expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

Base editing is a novel technology for engineering DNA in cells, which the potential to correct certain errors or mutations in the DNA, or inactivate disease-causing genes. Compared with currently available gene editing methodologies such as conventional CRISPR/Cas9, which creates "cuts" in the gene that can lead to adverse or negative effects, this new technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

Dr. Jonathan Frampton, Corporate Development Partner, Horizon Discovery, said: "The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialization. Horizon is pleased to offer an effective and precise base editing technology and, alongside Rutgers, aims to make base editing available to all appropriate cell and gene therapy companies as well as research departments. Partnering with leading organizations will help us to drive innovation and deliver the best therapy for the patient."

Dr. Shengkan 'Victor' Jin of Rutgers University stated: "The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies such as gene modified cells for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, over-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation. Other applications could include use as gene therapies for inherited genetic diseases including antitrypsin deficiency and Duchenne muscular dystrophy. In addition, we intend to take full advantage of the unique modularity and versatility features of Pin-point platform and develop efficient gene inactivation agents for potential treatment of many devastating diseases where the leading causal contributing factors are well defined. At the top of this disease list are Alzheimers disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia."

Dr. S. David Kimball, Senior Vice President for Research and Economic Development at Rutgers University, added: "The gene editing technology developed by Rutgers has the potential to revolutionize how scientists think about their search for better options and outcomes in the treatment of disease. It has the potential to solve some of the most persistent global health challenges. This partnership with Horizon Discovery is paving the way to deliver biotherapies for precision medicine and diagnostics and improve human health. I am proud that Rutgers, together with Horizon, is among the frontrunners in the field of gene editing."

View source version on businesswire.com: https://www.businesswire.com/news/home/20200114005331/en/

Contacts

Zyme Communications (Trade and Regional Media) Lorna CuddonTel: +44 (0)7811 996 942Email: lorna.cuddon@zymecommunications.com

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Horizon Discovery to Provide Access to Novel Base Editing Technology - Yahoo Finance

Recommendation and review posted by Bethany Smith

XconomyBoston

JP Morgans annual healthcare conference convenes in San Francisco this week and Generation Bio CEO Geoff McDonough is betting gene therapy will be among the discussion topics. While such therapies now offer ways to address some rare diseases that previously had no treatment, they also have limitations.

Generation Bio is among the new wave of companies trying to avoid or overcome the challenges facing the current slate of gene therapies. To support its efforts, the Cambridge, MA-based company has raised an additional $110 million for its most advanced programs while also aiming for something more.

It gets us to the clinic, although its almost certain well take the company public ahead of that, McDonough says.

The Series C financing includes crossover investors, firms that back both private and public companies and whose involvement is viewed as a sign a company is preparing to go public. T. Rowe Price led Generation Bios latest round, joined by Farallon, and Wellington Management Company. Also participating in the round were earlier investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital, and an entity associated with SVB Leerink. The latest round comes two years after Generation Bio closed a $100 million Series B round.

Currently available gene therapies (and some that are still in development) use an engineered virus to deliver their genetic payloads. But the virus can be a problem. Some people have preexisting antibodies to adeno-associated virus (AAV), the virus used in many gene therapies. And if patients dont already have those antibodies, they can develop them after being dosed with the treatment. That means patients cant receive another dose if they need it, and so far, the durability of gene therapies is not yet known.

Generation Bio avoids viral delivery altogether. The companys gene therapies use closed-ended DNA, or ceDNA, packaged inside a lipid nanoparticle. McDonough says it works just like AAV and it has the added advantage of being easier and less expensive to manufacture compared to AAV-based gene therapies. He adds that if patients need another dose, antibodies wont be a problem. But the company still needs to show that in clinical trials.

Generation Bios most advanced programs are for the blood disorder hemophilia A and phenylketonuria (PKU), an inherited metabolic disorder. There are other companies developing gene therapies for both diseases. Pfizer (NYSE: PFE) has taken over development of SB-525, a hemophilia A gene therapy candidate initially developed by its partner, Sangamo Therapeutics (NASDAQ: SGMO). BioMarin Pharmaceutical (NASDAQ: BMRN) on Monday announced that US and UK regulators cleared the company to begin clinical testing of BMN 307, its experimental gene therapy for PKU. Both experimental treatments use AAV to deliver the therapies to cells.

Besides supporting its hemophilia A and PKU clinical research, McDonough says the new financing will enable his company to look at other rare metabolic disorders such as Wilson and Gaucher disease. He adds that the company will also explore how its technology can be used to deliver therapies to more tissues in the body, such as skeletal tissue and the retina. By being able to reach more tissues, the goal is to expand the scope of gene therapy beyond rare diseases and bring it to the masses, he explains.

McDonough says Generation Bio has been careful not to work with other companies because its technology was still in development. But now that the technology is more mature, the biotech will explore research alliances with larger pharmaceuticalfirms.

I think we will do a partnership this year, he says. The question is in what domain and with which partner.

Photo by Jeremy Bishop on Unsplash

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan [[at]] xconomy.com.

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Generation Bio Pockets $110M for Next Wave of Gene Therapy, IPO Plans - Xconomy

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NEW YORK and GAINESVILLE, Fla., Jan. 13, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) and AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich's ataxia (FA), today announced the award of MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio's phase 2 clinical trial of a gene-replacement therapy for the disease.

MVP is MDA's drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular disorders. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics.

FA is a rare, hereditary disease that causes damage to parts of the spinal cord and brain, and there is currently no cure. AavantiBio was founded with the aim of developing an effective treatment for the disease and improving the lives of FA patients.

"With the approval of several first-time gene-targeting therapies for neuromuscular diseases over the past several years, including the first-ever gene-replacement therapy for spinal muscular atrophy, our community should have much to hope for as more and more therapies continue to be developed," says Lynn O'Connor Vos, MDA's president and CEO. "MDA is thrilled to be a part of the quest to help further develop the first gene-targeting therapy for the treatment of Friedreich's ataxia. By partnering with AavantiBio, together we can address the unmet need faced by patients who live with this genetic disease, for which there are still no treatments and no cures."

Co-founders Manuela Corti, PT, PhD, assistant professor of Pediatrics at the University of Florida, Gainesville, and Barry Byrne, MD, PhD, associate chair of Pediatrics and director of the Powell Gene Therapy Center at UF, started working with the FA community five years ago and are thrilled about their new partnership with MDA.

"This is a great opportunity for AavantiBio, and we're thankful to the MDA for their generous contribution," Dr. Corti says. "We hope to strengthen our collaboration as we work together on this project."

Dr. Corti and Dr. Byrne aim to include both adults and children who have FA in their clinical trials, paving the way for new solutions.

"We're delighted to take the next step in fulfilling our commitment to patients and families living with FA based on this investment from MDA," Dr. Byrne says. "We're looking forward to initiating screening for the first clinical study and a pivotal study in FA before the end of the year. The endorsement and investment from the MDA will be key to our programmatic growth."

MDA's investment will help accelerate AavantiBio's mission and begin production of the clinical gene vector for its therapy program. Clinical trials are expected to begin in 2020.

Dr. Corti was previously awarded an MDA research grant to develop and test a gene-replacement therapy in a mouse model of FA. With the current funding, AavantiBio will sponsor a study at the University of Florida led by Sub Subramony, MD, professor of Neurology. The clinical trial will assess changes in neurological and cardiac function in patients with FA treated with both intravenous (systemic) and intrathecal (into-the-spine) injections of the company's gene-replacement therapy for the mutated FXN gene.

About Friedreich's ataxia

FA is a mitochondrial disease. Mutations in the frataxin gene (FXN) lead to decreased production of the frataxin protein, resulting in diminished energy production in cells, including those of the nervous system and heart. FA's major neurological symptoms include muscle weakness and ataxia, or a loss of balance and coordination. FA mostly affects the spinal cord and the peripheral nervesthat connect the spinal cord to the body's muscles and sensory organs, but it can also affect the cerebellum (causing ataxia) and heart. The prevalence of FA has been estimated at 1 in every 50,000 individuals worldwide. Symptoms typically begin between the ages of 5 and 15 years, and the rate of progression varies from person to person. There currently are no effective cures or treatments for FA.

About AavantiBio

Founded in 2017 and based inGainesville, Fla., AavantiBio is a biotechnology companyfounded on the vision of creating the first effective treatment for FA. The company's gene-replacement therapy approach uses an adeno-associated virus (AAV) vector to deliver a functional copy of the FXN gene to a patient's cells.

The co-founders bring more than 30 years of research experience in the field of neuromuscular disease, including their current work focusing on FA. Dr. Corti has more than 10 years of research experience in gene therapy approaches for the treatment of Duchenne muscular dystrophy and Pompe disease. Dr. Byrne has made significant contributions to theunderstanding and treatment of Pompe disease. He was previously awarded nearly $2 million in MDA funding to conduct foundational research in developing and testing AAV vectors in animal models of muscular dystrophy, and he was the first to show that AAV vectors are able to effectively express therapeutic genes in striated muscle cells.

About the Muscular Dystrophy Association

MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to approved, life-changing therapies across multiple neuromuscular diseases. We support the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions, and each year thousands of children and young adults learn vital life skills and gain independence at MDA Summer Camp and through recreational programs. For more information visitmda.org.

View original content to download multimedia:http://www.prnewswire.com/news-releases/mda-awards-venture-philanthropy-funding-of-more-than-1m-to-aavantibio-to-develop-gene-targeting-therapy-for-friedreichs-ataxia-300985256.html

SOURCE Muscular Dystrophy Association

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MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich's Ataxia - P&T Community

Recommendation and review posted by Bethany Smith

The acquisition gives Veristat control of The Clinical Trial Company, a UK-based contract research organization (CRO) known by the abbreviation TCTC Group.

TCTC Group works out of two sites in the UK, a headquarters near Manchester, in northern England, and a specialist central nervous system (CNS) site in Scotland. Following the takeover, the two UK sites have become Veristats sole presence in Europe.

In North America, Veristat has positioned itself as a CRO focused on the needs of emerging, small and mid-sized biopharma companies. That positioning has led Veristat to have a role in a significant minority of drug development projects.

Across 2018, more than 10% of the drugs approved by the US Food and Drug Administration (FDA) had benefited from the support of Veristat. The FDA approvals brought the number of regulatory submissions supported globally by Veristat up above 75.

Having bought TCTC Group, Veristat is now positioned to spread the approach that brought it these successes to a new continent.

Explaining the motivation for the takeover, Veristat CEO Patrick Flanagan said, With the addition of TCTC Group, Veristat has gained a highly skilled team of experts to support our clients worldwide. Providing superior clinical services with our expanded team will enable us to better manage, recruit and run our clients trials on a global scale.

TCTC Groups CNS expertise also factored into Flanagans thinking. The Veristat CEO pointed to the industrys increased funding in CNS and gene therapy research to explain why he sees the field as a critical therapeutic category.

CNS is part of Veristats traditional breadth of focus. Most of the US CROs clinical trial work covers cancers, rare diseases, neurological disorders, cardiovascular disease and infectious diseases.

TCTC Group built up its expertise in the CNS field over the course of its 17-year history as an independent company, going as far as to create a dedicated division called The CNS Company.

More broadly, TCTC Group focused on products without a classical road map to approval. That focus led TCTC Group to work on advanced therapy medicinal products, such as cell and gene therapies, as well as orphan medicines and drug-device combinations.

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CRO Veristat buys TCTC Group to expand in Europe - OutSourcing-Pharma.com

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced that it has appointed John C. Martin, Ph.D., to its Board of Directors, effective today. Dr. Martin brings decades of executive leadership to Sareptas board, having played an instrumental role in building one of the worlds foremost biotechnology companies. During his 20-year tenure as chief executive officer of Gilead Sciences, he oversaw the growth of the company and development of its scientific portfolio into 24 marketed products.

Johns business record is unparalleled, and he brings expertise and insight gleaned from leading the long-term, transformational growth of one of the industrys premier companies that will add to the strength of the Sarepta board. We are incredibly fortunate to welcome him to the companys board of directors, saidM. Kathleen Behrens, Ph.D., Chairperson of Sareptas Board of Directors.

Having introduced curative therapies, Johns vision, acumen, and wisdom are unique in our industry. As Sarepta prepares to deliver one-time therapies for rare diseases, Johns strategic guidance as a member of the companys board will be invaluable as we work to change the model for how rare diseases are treated, said Doug Ingram, Sareptas president and chief executive officer.

Dr. Martin served as the Executive Chairman and Chairman of Gilead Sciences, Inc., from March 2016 through March 2019, after having served as Chairman and Chief Executive Officer from June 2008 through March 2016 and President and Chief Executive Officer from 1996 through May 2008. He joined Gilead in 1990 as Vice President, Research and Development. Prior to Gilead, Dr. Martin held several leadership positions at Bristol-Myers Squibb and Syntex Corporation. Martin currently serves on the Board of Directors at The Scripps Research Institute and Kronos Bio. Additionally, he served on the Centers for Disease Control/Health Resources and Services Administrations Advisory Committee on HIV and STD Prevention and Treatment and was a member of the Presidential Advisory Council on HIV/AIDS. In 2008, Dr. Martin was inducted into the National Academy of Engineering and, in 2019, he received the National Academy of Sciences Award for Chemistry in Service to Society.

Dr. Martin holds a Ph.D. in organic chemistry from the University of Chicago, an MBA from Golden Gate University and a B.S. degree in chemical engineering from Purdue University.

Heidrick & Struggles led the search process for Sarepta.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Forward-Looking StatementsThis press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the expectation that Dr. Martins expertise and insight will add to the strength of the Sarepta board and that Dr. Martins strategic guidance will be invaluable as Sarepta prepares to deliver one-time therapies for rare diseases; and Sareptas mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: Sarepta may not be able to execute on its business plans, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its products to U.S. and ex-U.S. markets for various reasons including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2018 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2018 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

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

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

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

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Sarepta Therapeutics Appoints Renowned Biotech Executive John C. Martin to its Board of Directors - GlobeNewswire

Recommendation and review posted by Bethany Smith

NEW YORK, Jan. 13, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium"), announced today that it has entered into an agreement with the University of California, Davis (UC Davis) to utilize Actinium's Antibody Radiation-Conjugate or ARC apamistamab-I-131 for targeted conditioning and replace the chemotherapy conditioning being used in an ongoing Phase 1/2 stem cell gene therapy clinical trial. In the trial, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. This is the first gene therapy clinical trial that will utilize ARC based conditioning. The clinical trial will be conducted at UC Davis and may be expanded to additional sites in the future.

Dr. Mehrdad Abedi, Professor, Hematology and Oncology at UC Davis and study lead, said, "This collaboration represents an exciting combination of revolutionary technologies that could further our ability to treat patients with HIV and other life-threatening diseases with gene therapy. Despite the advances made in the field of gene therapy, the reliance on non-targeted chemotherapy and external radiation as conditioning regimens is less than optimal and poses a problem that we hope to reduce or eliminate as part of this collaboration by replacing our conditioning regimen in this study with Actinium's ARC based targeted conditioning. Advances in HIV therapies have dramatically improved patient survival, but current therapies require life-long daily use to keep the HIV virus at bay, can have severe side effects, may be overcome by HIV resistance and do not address the needs of all patients like those in this study with HIV-related lymphomas. We envision a future where a single treatment of our stem cell gene therapy can cure patients of their lymphoma and HIV leaving the patient with a new immune system that can fight, be resistant to and prevent the mutation of HIV. Apamistamab-I-131's demonstrated antitumor effect against lymphoma and ability to condition patients in a targeted manner with a demonstrated tolerable safety profile in the bone marrow transplant setting makes it an ideal conditioning agent for this patient population. Based on these factors and extensive supporting clinical data in the Iomab-B program, we selected this ARC as the conditioning agent for the next phase of our trial as we believe antibody radiation-conjugates are more advanced and hold distinct advantages over novel but unproven conditioning technologies such as Antibody Drug Conjugates and naked antibodies that are beginning to be developed albeit at the preclinical stage."

In the current clinical trial, the anti-HIV stem cell gene therapy is produced by taking a patient's own or autologous, blood forming stem cells and genetically modifying them via gene therapy with a combination of three anti-HIV genes. The intended result is for the gene modified bone marrow stem cells to produce a new immune system and newly arising immune cells that are resistant to HIV via a single treatment. Conditioning is necessary prior to adoptive cell therapies such as gene therapy to eliminate certain cell types such as immune cells and stem cells in the bone marrow so the transplanted cells can engraft. Until now, conditioning in this trial, as is typical, used a multi-drug chemotherapy regimen administered over several days. This approach is non-targeted, associated with toxicities that impairs patients and restricts the use and efficacy of cellular therapy. Apamistamab-I-131, which requires just one therapeutic administration, will displace the non-targeted chemotherapy to condition patients in a targeted manner with the goal of reducing conditioning related toxicities and improving patient outcomes. Actinium and UC David will cross-reference their respective Investigational New Drug applications and will work collaboratively to obtain necessary regulatory and institutional approvals. In this clinical collaboration, Actinium will provide drug product, support for its administration and certain trial costs. UC Davis will be responsible for the production of the anti-HIV stem cell gene therapy and overall conduct of the study and its cost.

Dr. Dale Ludwig, Actinium's Chief Scientific Officer, said, "We are excited to be working with Dr. Abedi on this clinical study and we appreciate his recognition of the value of our Iomab-ACT targeted conditioning program may provide in support of gene stem cell therapy. This targeted approach using our CD45 ARC, enables both anti-tumor activity and effective conditioning with the potential for reduced toxicity compared to non-targeted chemotherapy and external radiation in the bone marrow transplant setting. Supported by extensive clinical investigation in 12 trials and over 300 patients, a single therapeutic dose of apamistamab-I-131 is sufficient for conditioning and, due to its dual activity, even a patient with active disease could expect to receive therapy within two weeks, which is anticipated to lead to better outcomes compared to chemotherapy, external beam radiation, or exploratory approaches such as naked antibodies or Antibody Drug Conjugates. In addition, CD45, the target of apamistamab-I-131, is ideal for targeted conditioning, as it is not expressed outside of the haemopoietic system and, because it is a poorly internalizing receptor. An ARC approach which does not require internalization of its radionuclide warhead for target cell killing, is anticipated to be more viable and more effective than Antibody Drug Conjugate approaches which need to internalize their payloads. Given the potential of this ARC targeted conditioning technology for bone marrow transplant, we are grateful to Dr. Abedi for the opportunity to advance the Iomab-ACT program into the promising field of gene stem cell therapy."

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "Actinium is thrilled to be working with UC Davis and honored to now be part of this important trial. It has become evident that better conditioning regimens are needed for cell and gene therapies to reach their full potential. Our team is proud to be the first company to establish a clinical stage targeted conditioning portfolio for both cell and gene therapy. We are pleased to extend our ARC technology for targeted conditioning into these rapidly advancing fields and we are committed to establishing a strong leadership position in enabling these adoptive cell therapies fully realize their great potential for improving patients' lives."

Apamistamab-I-131's demonstrated conditioning and antitumor effect in lymphoma1

Actinium's apamistamab-I-131 ARC has been studied as a targeted conditioning agent in over 300 patients in the bone marrow transplant setting in the Iomab-B Program and is currently being studied in a pivotal Phase 3 clinical (SIERRA) trial in patients with relapsed or refractory acute myeloid leukemia. Clinical proof of concept has been established with Iomab-B for targeted conditioning in high-risk, relapsed or refractory lymphoma patients prior to an autologous stem cell transplant where a favorable safety profile with no dose limiting toxicities and minimal non-hematologic toxicities observed and promising efficacy with median overall survival not reached (range: 29 months to infinity) and 31% of patients in prolonged remission at a median of 36 months follow up (range: 25 41 months)1.

1) Cassaday et al. Phase I Study of a CD45-Targeted AntibodyRadionuclide Conjugate for High-Risk Lymphoma. AACR Clin Cancer Res Published OnlineFirst September 3, 2019

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, apamistamab-I-131 (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Apatmistamamb-I-131 will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related...

Recommendation and review posted by Bethany Smith

White blood cells circulate around the blood and help the immune system fight off infections.

Stem cells in the bone marrow are responsible for producing white blood cells. The bone marrow then stores an estimated 8090% of white blood cells.

When an infection or inflammatory condition occurs, the body releases white blood cells to help fight the infection.

In this article, learn more about white blood cells, including the types and their functions.

Health professionals have identified three main categories of white blood cell: granulocytes, lymphocytes, and monocytes. The sections below discuss these in more detail.

Granulocytes are white blood cells that have small granules containing proteins. There are three types of granulocyte cells:

These white blood cells include the following:

Monocytes are white blood cells that make up around 28% of the total white blood cell count in the body. These are present when the body fights off chronic infections.

They target and destroy cells that cause infections.

According to an article in American Family Physician, the normal range (per cubic millimeter) of white blood cells based on age are:

The normal range for a pregnant women in the 3rd trimester is 5,80013,200 per cubic millimeter.

If a person's body is producing more white blood cells than it should be, doctors call this leukocytosis.

A high white blood cell count may indicate the following medical conditions:

Surgical procedures that cause cells to die can also cause a high white blood cell count.

If a person's body is producing fewer white blood cells than it should be, doctors call this leukopenia.

Conditions that can cause leukopenia include:

Doctors may continually monitor white blood cells to determine if the body is mounting an immune response to an infection.

During a physical examination, a doctor may perform a white blood cell count (WBC) using a blood test. They may order a WBC to test for, or rule out, other conditions that may affect white blood cells.

Although a blood sample is the most common approach to testing for white blood cells, a doctor can also test other body fluids, such as cerebrospinal fluid, for the presence of white blood cells.

A doctor may order a WBC to:

The following are conditions that may impact how many white blood cells a person has in their body.

This is a condition wherein a person's body destroys stem cells in the bone marrow.

Stem cells are responsible for creating new white blood cells, red blood cells, and platelets.

This is an autoimmune condition wherein the body's immune system destroys healthy cells, including red and white blood cells.

HIV can decrease the amount of white blood cells called CD4 T cells. When a person's T cell count drops below 200, a doctor might diagnose AIDS.

Leukemia is a type of cancer that affects the blood and bone marrow. Leukemia occurs when white blood cells rapidly produce and are not able to fight infections.

This condition causes a person's body to overproduce some types of blood cells. It causes scarring in a person's bone marrow.

Whether or not a person needs to alter their white blood cell count will depend on the diagnosis.

If they have a medical condition that affects the number of white blood cells in their body, they should talk to a doctor about the goals for their white blood cell count, depending on their current treatment plan.

A person can lower their white blood cell count by taking medications such as hydroxyurea or undergoing leukapheresis, which is a procedure that uses a machine to filter the blood.

If a person's white blood cell count is low due to cancer treatments such as chemotherapy, a doctor may recommend avoiding foods that contain bacteria. This may help prevent infections.

A person can also take colony-stimulating factors. These may help prevent infection and increase the number of white blood cells in the body.

White blood cells are an important part of the body's immune system response. There are different types of white blood cell, and each has a specific function in the body.

Certain conditions can affect the number of white blood cells in the body, causing them to be too high or too low.

If necessary, a person can take medication to alter their white blood cell count.

More here:
White blood cells: Function, ranges, types, and more - Medical News Today

Recommendation and review posted by Bethany Smith

BOSTON Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.

The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.

It was a critical moment in medical science.

For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the diseases horrifying hallmark: episodes of agony that begin in babyhood.

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts said the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African American, often poor or of modest means.

The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.

This is the story of two quests for a sickle cell cure one by the Obando family and one by a determined scientist at Boston Childrens Hospital, Dr. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s.

Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee Obando. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood.

Scientists have been experimenting with gene therapy for two decades, with mixed success. And it will be years before they know if this new procedure is effective in the long term. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.

Four weeks after the infusion of stem cells, Helen was strong enough to be discharged. At home, in Lawrence, Massachusetts, on a sofa with her mother by her side, she put a hand over her eyes and started to sob. She and her family wondered: Would it work? Was her suffering really over?

A Familys Nightmare

Sheila Cintron, 35, and Byron Obando, 40, met when she was in the eighth grade and he was a high school senior. They fell in love. Haylee, their first child, was born in 2001, when Cintron was 17.

When a newborn screening test showed that Haylee had the disease, her father asked, Whats sickle cell?

They soon found out.

As the family gathered for her first birthday party, Haylee started screaming inconsolably. They rushed her to the hospital. It was the first of many pain crises.

Doctors warned the parents that if they had another baby, the odds were 1 in 4 that the child would have sickle cell, too. But they decided to take the chance.

Less than two years later, Helen was born. As bad as Haylees disease was, Helens was much worse. When she was 9 months old, a severe blockage of blood flow in her pelvis destroyed bone. At age 2, her spleen, which helps fight bacterial infections, became dangerously enlarged because of blocked blood flow. Doctors surgically removed the organ.

After Helen was born, her parents decided not to have any more children. But four years later, Cintron discovered she was pregnant again.

But they were lucky. Their third child, Ryan Obando, did not inherit the sickle cell mutation.

As Ryan grew up, Helens health worsened. When he was 9, Helens doctors suggested a drastic solution: If Ryan was a match for her, he might be able to cure her by giving her some of his bone marrow, though there would also be major risks for her, including death from severe infections or serious damage to organs if his immune system attacked her body.

As it turned out, Ryan matched not Helen but Haylee.

The transplant succeeded, but her parents asked themselves how they could stand by while one daughter was cured and the sicker one continued to suffer.

There was only one way to get a sibling donor for Helen: have another baby. In 2017, the couple embarked on another grueling medical journey.

Obando had a vasectomy, so doctors had to surgically extract his sperm from his testicles. Cintron had 75 eggs removed from her ovaries and fertilized with her husbands sperm. The result was more than 30 embryos.

Not a single embryo was both free of the sickle cell gene and a match for Helen.

So the family decided to move to Mesa, Arizona, from Lawrence, where the cold, which set off pain crises, kept Helen indoors all winter. The family had already sold their house when they heard that doctors at Boston Childrens were working on sickle cell gene therapy.

Cintron approached Dr. Erica Esrick, a principal investigator for the trial. But the trial wasnt yet open to children.

Figuring Out the Science

Nothing had prepared Orkin for the suffering he witnessed in his 30s as a medical resident in the pediatric hematology ward at Boston Childrens. It was the 1970s, and the beds were filled with children who had sickle cell crying in pain.

Orkin knew there was a solution to the puzzle of sickle cell, at least in theory: Fetuses make hemoglobin the oxygen-carrying molecules in blood cells with a different gene. Blood cells filled with fetal hemoglobin do not sickle. But the fetal gene is turned off after a baby is born, and an adult hemoglobin gene takes over. If the adult gene is mutated, red cells sickle.

Researchers had to figure out how to switch hemoglobin production to the fetal form. No one knew how to do that.

Orkin needed ideas. Supported by the National Institutes of Health and Howard Hughes Medical Institute, he kept looking.

The breakthrough came in 2008. The cost of gene sequencing was plummeting, and scientists were finding millions of genetic signposts on human DNA, allowing them to home in on small genetic differences among individuals. Researchers started doing large-scale DNA scans of populations, looking for tiny but significant changes in genes. They asked: Was there a molecular switch that flipped cells from making fetal to adult hemoglobin? And if there was, could the switch be flipped back?

They found a promising lead: an unprepossessing gene called BCL11A.

In a lab experiment, researchers blocked this gene and discovered that the blood cells in petri dishes started making fetal instead of adult hemoglobin.

Next they tried blocking the gene in mice genetically engineered to have human hemoglobin and sickle cell disease. Again, it worked.

Patients came next, in the gene therapy trial at Boston Childrens that began in 2018.

The trial run by Dr. David Williams, an expert in the biology of blood-forming stem cells at Boston Childrens, and Esrick has a straightforward goal: Were going to reeducate the blood cells and make them think they are still in the fetus, Williams said.

Doctors gave adult patients a drug that loosened stem cells immature cells that can turn into red blood cells from the bone marrow, their normal home, so they floated free in the bloodstream. Then they extracted those stem cells from whole blood drawn from the patient.

The researchers used a disabled genetically engineered AIDS virus to carry information into the stem cells, flipping on the fetal hemoglobin gene and turning off the adult gene. Then they infused the treated stem cells into patients veins. From there, the treated cells migrated into the patients bone marrow, where they began making healthy blood cells.

With the success in adults, the Food and Drug Administration said Boston Childrens could move on to teenagers.

When her mother told her about the gene therapy trial, Helen was frightened. But the more she thought about it, the more she was ready to take the risk.

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared.

Helen was scheduled for her six-month checkup Dec. 16. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved, even with blood transfusions. She had no signs of sickle cell disease.

More here:
At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens - Boston.com

Recommendation and review posted by Bethany Smith

The Cord Blood Stem Cells Market is predicted to worth +6500 Million USD with a CAGR of +30% over the forecast period 2020 to 2025.

Three sources of stem cells are bone marrow, peripheral blood, and cord blood. The blood in the umbilical cord is called cord blood and is collected at the time of delivery. Cord blood is an abundant source of Red Blood Cells (RBCs), white blood cells (WBCs), platelets and hematopoietic stem cells, and is extracted and stored in a private blood bank for the purpose of treating the disease in the future as needed.

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The global Cord Blood Stem Cells Market analytical report has recently published by Report Consultant to its massive repository. The research report has been summarized with informative and technical details of the dynamics of the market. It has been compiled by using some significant research methodologies such as primary and secondary research techniques. The report also elaborates on the factors which are fueling or hampering the growth of the market. It gives more focus on recent trends and technologies which are boosting the performance of the companies.

Cord Blood Stem Cells Market Key Players:

Cord Blood America Inc, Cryo-Cell International Inc, Cryo-Save AG, Cord Blood Registry Systems Inc, Viacord Inc, China Cord Blood Corporation, Cordlife Group Ltd, Vita 34 AG, Lifecell International Pvt. Ltd, Stemcyte Inc

The Cord Blood Stem Cells Market is segmented by means of storage service, application, and region.

Storage service: Public cord blood bank and Private cord blood bank

Cord blood stem cell market segmentation by application: Blood disease, Cancer, Acute leukemia, Krabbe diseases, and other diseases

Regions: North America (USA, Canada, Mexico), Europe (Germany, France, UK, Italy, Russia), Asia Pacific (China, India, Japan, South Korea, Australia, Indonesia, Malaysia), Middle East and Africa (Bahrain, Egypt, Jordan, Kuwait, Morocco, Oman, Qatar, Saudi Arabia, Syria)

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The report Describes the Cord Blood Stem Cells Market basics like definitions, classifications, applications and industry chain overview, industry policies and plans, product specifications, manufacturing processes, cost structures and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, capacity utilization, supply, demand and industry growth rate, etc. In the end, the report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

Global research Cord Blood Stem Cells Market report highlights:

Original post:
What is Cord Blood Stem Cells Market and What Factors will drive the Industry including Leading Players Cord Blood America Inc, Cryo-Cell...

Recommendation and review posted by Bethany Smith

2020 Honorees include Cystic Fibrosis Foundation, Emily Whitehead Foundation, Gift of Life Marrow Registry and Ret. Major General Bernard Burn Loeffke (US Military)

Miami, FL, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The formal ceremony of the 2020 Stem Cell and Regenerative Medicine Action Awards will take place at a gala reception and dinner on January 23, during the 15th annual World Stem Cell Summit (WSCS) at the Hyatt Regency in Miami. Since 2005, the nonprofit Regenerative Medicine Foundation (RMF) (formerly Genetics Policy Institute) has recognized the stem cell and regenerative medicine community's leading innovators, leaders, and champions through its annual awards reception.

Bernard Siegel, Executive Director of Regenerative Medicine Foundation and founder of the World Stem Cell Summit, said, The 2020 Action Awards will recognize three important organizations that are positively impacting the emerging field of regenerative medicine. We will also honor a retired Major General, who has capped off his military and diplomatic career by promoting the cause of world peace through medicine. All of these distinguished honorees will be recognized for their devotion to improving health and developing cures through advocacy, innovation, leadership and inspiration. In addition, the wounded warrior veterans community of South Florida will also receive special recognition at the event.

Meet the 2020 Stem Cell & Regenerative Medicine Action Award Honorees:

Innovation Award: With the motto, We will not rest until we find a cure, the Cystic Fibrosis Foundation is geared towards the successful development and delivery of treatments, therapies and a cure for every person with cystic fibrosis. CF Foundation has added decades to the lives of people with the disease as a direct result of advances in treatment and care made possible through its innovative business model- venture philanthropy. The Foundation recently unveiled its Path to a Cure research agenda aimed at addressing the root genetic cause of the disease and is currently funding industry programs aimed at gene delivery with the goal of progressing into clinical studies in 2021.

Inspiration Award: Emily Whitehead Foundation is a nonprofit organization committed to raising funds to invest in the most promising pediatric cancer research. Tom and Kari Whitehead founded EWF in honor of their daughter Emily, the first child in the world to receive CAR T-cell therapy, training her own cells to fight cancer. Her inspiring story focused public attention on thepotential for cancer immunotherapy to transform cancer treatment,as well as the need to support lifesaving cancer immunotherapy research. The foundation provides support to pediatric cancer patients and promotes awareness of the disease through education and sharing other inspiring stories.

Advocacy Award: Gift of Life Marrow Registry was established in 1991 by Jay Feinberg and his family after Jay received a life-saving bone marrow transplant. Gift of Life is dedicated to saving lives and facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, sickle cell and other diseases. In 2019, Gift of Life opened the worlds first apheresis center fully integrated within a registry, the Dr. Miriam and Sheldon G. Adelson Gift of Life-Be The Match Stem Cell Collection Center. With the collection center and rapidly expanding donor database, Gift of Life will launch a biobank to advance cellular therapies using allogeneically sourced cells in 2020.

Leadership Award: Ret. Major General Bernard Burn Loeffke, PhD (US Military) is a highly decorated Special Forces officer, diplomat and medical officer.He survived two helicopter crashes and was wounded in combat. After the Vietnam War, he served as the Army Attach at theU.S. Embassy in Moscow, first Defense Attach at the U.S Embassy in Beijing, a staff officer in theWhite House, and Director of the Commission onWhite House Fellows. His last command was Commanding General of Army South. After 35 years in the military, he became a medical officer traveling the world on relief missions to third and fourth world countries. Presently, at age 85, he champions the hydrocephalus and wounded warrior communities. He continues to serve as an inspiration and supporter of building peaceful international relations through medical partnerships and played a pivotal role as a keynote speaker at the inaugural 2019 World Stem Cell Summit CHINA.He is called the Peace General in Latin America. In China, he is simply known as The General, our Friend.

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To learn more about past honorees and details for sponsoring or attending the upcoming 2020 Stem Cell and Regenerative Medicine Action Awards dinner, please visit, https://www.worldstemcellsummit.com/stem-cell-action-awards/

About the World Stem Cell Summit (WSCS)

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About the Regenerative Medicine Foundation (RMF)

The nonprofit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF unites the worlds leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations. We maintain a trusted network of leaders and pursue our mission by producing our flagship World Stem Cell Summit series of conferences and public days, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, supporting our official journal partner STEM CELLS Translational Medicine (SCTM), promoting solution-focused policy initiatives both nationally and internationally and creating STEM/STEAM educational projects. For more information about RMF, please visit: http://www.regmedfoundation.org.

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Joseph DawsonRegenerative Medicine Foundation561-906-4755joseph@regmedfoundation.org

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami - Yahoo Finance

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What are the most common types of brain tumors in children, and what treatment options are available?

Brain tumors are the most common solid tumors affecting children, with approximately 4,500 new cases each year in the U.S.

As brain tumors expand or block the normal pathways in the brain, the pressures inside the skull expand. As a result, symptoms of brain tumors can include headaches, seizures, lethargy, nausea and vomiting. A child experiencing progressively worsening symptoms like these should be evaluated by a pediatrician or in the emergency room. The doctors evaluation may include a scan of the brain. If the scan shows a tumor, the next step is a consultation with a neurosurgeon.

The majority of pediatric brain tumors occur in the posterior fossa (located near the bottom of the skull and the brain stem). The most common tumors include medulloblastoma, pilocytic astrocytoma, and ependymoma. Other less common tumors can occur in the cerebral hemispheres (the two main portions of the brain) and include astrocytomas, gangliogliomas, craniopharyngiomas, and germ cell tumors.

Surgery is usually the first step in treatment when a brain tumor is discovered. The goals of surgery are to determine whether the tumor is cancerous and remove all or as much of the tumor as safely as possible. At UVa Childrens Hospital, the latest technologies are utilized to help perform surgery, including intraoperative MRI, navigation, ultrasound and minimally invasive endoscopic surgery. Based on the types of cells found in the brain tumor, additional treatments may be needed. These therapies may include chemotherapy, radiation therapy, proton therapy, stem cell rescue and bone marrow transplantation and/or supportive care for rehabilitation.

More recent treatment options have focused on precision medicine and targeted drug therapy. Targeted drug treatments can cause brain tumor cells to die by blocking abnormalities present within these cells. These drugs are changing how brain tumors are treated while improving outcomes. Current research is focused on understanding the molecular basis of tumor formation and discovery of new targets for treatment.

At UVa, we are committed to providing the best neurosurgical care for children through our multidisciplinary brain tumor team, consisting of neurosurgery, neurology, pediatric oncology and radiation oncology.

For more information, visit childrens.uvahealth.com/services/pediatric-neurosurgery.

Dr. Hasan R. Syed and Dr. John Jane Jr. are pediatric neurosurgeons at UVa Childrens Hospital.

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Ask the Expert: What are the most common types of brain tumors in children? - The Daily Progress

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Roshen Confectionery Corporation will allocate UAH 50 million for the overhaul of the oncohematology department and the creation of an autologous bone marrow transplantation department at the National Cancer Institute.

According to the company's press release, the project will last almost two years.

"In the building where the oncohematology and chemotherapy department is located, the roof has been leaking for many years, water leaked from the sewer under the foundation as a result, almost all the walls of the building have a fungus that is simply deadly for people with this disease. In early autumn, Roshen began the overhaul of part of the premises of the second building of the National Cancer Institute. We plan to complete the work in August 2020," Iryna Ponomarenko, the director for social projects development at Roshen Confectionery Corporation, said.

In 2018, the corporation repaired and equipped a room intended for apheresis (collection) of stem cells (for bone marrow transplantation) and donor platelets for a total of UAH 2.9 million.

In total, in 2017-2018 Roshen invested UAH 357 million in social projects.

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Roshen will issue UAH 50 mln for development of National Cancer Institute - Interfax Ukraine

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Jasper Therapeutics, Inc., a Palo Alto, Calif.-based biotechnology company focused on hematopoietic cell transplant therapies, expanded its Series A financing with an additional investment of $14.1m.

The round was led by Roche Venture Fund with participation from other investors. This brought the total company financing to more than $50m to date.

The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

The company plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191.

Jasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies.

FinSMEs

09/01/2020

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Jasper Therapeutics Raises Additional $14.1M in Series A Financing - FinSMEs

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13 January 2020

Promising results from clinical trials give hope for using CRISPR/Cas9 genome editing to treat various heritable diseases and cancer in humans.

It has been seven years since the discovery that the CRISPR/Cas9 defence system, used by microbes to destroy viruses, could be re-engineered to edit the human genome. Since then researchers have carried out an array of experiments to explore potential applications.

Biophysist Dr He Jiankui sparked global controversy concerning the ethics of genome editing when he used CRISPR to genetically modify embryos, resulting in the birth of the first genome-edited babies (see BioNews 977).

Yet researchers worldwide have at the same time been investigating the use of CRISPR for non-heritable changes, modifying the genes in non-embryonic cells to treat a wide range of diseases.

'There's been a lot of appropriate caution in applying this to treating people, but I think we're starting to see some of the results of that work,' said Dr Edward Stadtmauer, a haematologist at the University of Pennsylvania, Philadelphia.

Over a dozen new clinical trials testing CRISPRtherapy on diseases such as cancer, HIV and sickle cell anaemia were listed on the clinicaltrials.gov database last year. One trial in its early stages used CRISPR to treat sickle cell anaemia and beta-thalassaemia, both genetic blood disorders that result in the production of an abnormal form of the oxygen-carrying protein, haemoglobin.

Two patients with these disorders were treated by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, using CRISPR to inactivate a gene that switches off the production of an alternative form of haemoglobin. Preliminary results of the study suggest that this therapy improved some of the symptoms but the participants will need to be followed for a longer period to be sure.

Results from two other trials, one in which genome-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancer, were less successful. In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University, Beijing, China and a lead author of the work.

Other researchers are trying to move beyond editing cells in vitro. In July 2019 a clinical trial was launched to treat Leber congenital amaurosis 10 (LCA10), a rare genetic disease that causes blindness. The trial, launched by two pharmaceutical companies, Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin, Ireland, will be the first trial that uses CRISPR to edit cells inside of the body. The researchers are testing AGN-151587 (EDIT-101), which is a novel CRISPR treatment delivered via adeno-associated virus (AAV) directly to the eye's light-sensing photoreceptor cells to remove the mutation that causes LCA10.

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The potential use of CRISPR to treat disease is gaining momentum - BioNews

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