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Friday, October 7, 2022

Adult brain's ability to recover vision

 A discovery about how some visually impaired adults could start to see offers a new vision of the brain's possibilities. The finding that the adult brain has the potential to partially recover from inherited blindness comes from a collaboration between researchers in the University of California, Irvine School of Biological Sciences and the School of Medicine. Their paper appears in Current Biology.

The team was examining treatment for Leber congenital amaurosis, known as LCA. The term refers to a group of inherited retinal diseases distinguished by severe visual impairment at birth. The condition, which stems from mutations in any of over two dozen genes, causes degeneration or dysfunction in the retina's photoreceptors.

Administering chemical compounds that target the retina, called synthetic retinoids, can restore a notable amount of vision in children with LCA. The UCI team wanted to find out if the treatment could make a difference for adults who have the condition.

"Frankly, we were blown away by how much the treatment rescued brain circuits involved in vision," said Sunil Gandhi, professor of neurobiology and behavior and the corresponding author. Gandhi is a fellow of UCI's Center for the Neurobiology of Learning and Memory and a member of the Center for Translational Vision Research. "Seeing involves more than intact and functioning retinae. It starts in the eye, which sends signals throughout the brain. It's in the central circuits of the brain where visual perception actually arises." Until now, scientists believed that the brain must receive those signals in childhood so that central circuits could wire themselves correctly.

Working with rodent models of LCA, the collaborators were surprised by what they found. "The central visual pathway signaling was significantly restored in adults, especially the circuits that deal with information coming from both eyes," Gandhi said. "Immediately after the treatment, the signals coming from the opposite-side eye, which is the dominant pathway in the mouse, activated two times more neurons in the brain. What was even more mind-blowing was that the signals coming from the same-side eye pathway activated five-fold more neurons in the brain after the treatment and this impressive effect was long-lasting. The restoration of visual function at the level of the brain was much greater than expected from the improvements we saw at the level of the retinae. The fact that this treatment works so well in the central visual pathway in adulthood supports a new concept, which is that there is latent potential for vision that is just waiting to be triggered."

The finding opens exciting research possibilities. "Whenever you have a discovery that breaks with your expectations about the possibility for the brain to adapt and rewire, it teaches you a broader concept," Gandhi said. "This new paradigm could aid in the development of retinoid therapies to more completely rescue the central visual pathway of adults with this condition."

Gandhi and first author Carey Huh, PhD, who initiated the project, teamed with Krzysztof Palczewski, Distinguished Professor of ophthalmology. Palczewski, director of the Center for Translational Vision Research, is renowned for his work on retinoids and the visual cycle. Philip Kiser, associate professor of physiology and biophysics, an expert on visual cycle biochemistry, helped lead the group. Kiser, who holds a joint appointment in ophthalmology, is a member, Center for Translational Vision Research.

The research was funded by the National Institutes of Health, the Department of Veterans Affairs and the Research to Prevent Blindness foundation.


Story Source:

Materials provided by University of California - IrvineNote: Content may be edited for style and length.


Journal Reference:

  1. Carey Y.L. Huh, Henri Leinonen, Taylor Nakayama, Julia R. Tomasello, Jianye Zhang, Jack Zeitoun, John P. Peach, Maximilian Halabi, Jianying Z. Kiser, Krzysztof Palczewski, Philip D. Kiser, Sunil P. Gandhi. Retinoid therapy restores eye-specific cortical responses in adult mice with retinal degenerationCurrent Biology, 2022; DOI: 10.1016/j.cub.2022.09.005

Protein partners that could heal heart muscle

 Scientists at the UNC School of Medicine have made a significant advance in the promising field of cellular reprogramming and organ regeneration, and the discovery could play a major role in future medicines to heal damaged hearts.

In a study published in the journal Cell Stem Cell, scientists at the University of North Carolina at Chapel Hill discovered a more streamlined and efficient method for reprogramming scar tissue cells (fibroblasts) to become healthy heart muscle cells (cardiomyocytes). Fibroblasts produce the fibrous, stiff tissue that contributes to heart failure after a heart attack or because of heart disease. Turning fibroblasts into cardiomyocytes is being investigated as a potential future strategy for treating or even someday curing this common and deadly condition.

Surprisingly, the key to the new cardiomyocyte-making technique turned out to be a gene activity-controlling protein called Ascl1, which is known to be a crucial protein involved in turning fibroblasts into neurons. Researchers had thought Ascl1 was neuron-specific.

"It's an outside-the-box finding, and we expect it to be useful in developing future cardiac therapies and potentially other kinds of therapeutic cellular reprogramming," said study senior author Li Qian, PhD, associate professor in the UNC Department of Pathology and Lab Medicine and associate director of the McAllister Heart Institute at UNC School of Medicine.

Scientists over the last 15 years have developed various techniques to reprogram adult cells to become stem cells, then to induce those stem cells to become adult cells of some other type. More recently, scientists have been finding ways to do this reprogramming more directly -- straight from one mature cell type to another. The hope has been that when these methods are made maximally safe, effective, and efficient, doctors will be able to use a simple injection into patients to reprogram harm-causing cells into beneficial ones.

"Reprogramming fibroblasts has long been one of the important goals in the field," Qian said. "Fibroblast over-activity underlies many major diseases and conditions including heart failure, chronic obstructive pulmonary disease, liver disease, kidney disease, and the scar-like brain damage that occurs after strokes."

In the new study, Qian's team, including co-first-authors Haofei Wang, PhD, a postdoctoral researcher, and MD/PhD student Benjamin Keepers, used three existing techniques to reprogram mouse fibroblasts into cardiomyocytes, liver cells, and neurons. Their aim was to catalogue and compare the changes in cells' gene activity patterns and gene-activity regulation factors during these three distinct reprogrammings.

Unexpectedly, the researchers found that the reprogramming of fibroblasts into neurons activated a set of cardiomyocyte genes. Soon they determined that this activation was due to Ascl1, one of the master-programmer "transcription factor" proteins that had been used to make the neurons.

Since Ascl1 activated cardiomyocyte genes, the researchers added it to the three-transcription-factor cocktail they had been using for making cardiomyocytes, to see what would happen. They were astonished to find that it dramatically increased the efficiency of reprogramming -- the proportion of successfully reprogrammed cells -- by more than ten times. In fact, they found that they could now dispense with two of the three factors from their original cocktail, retaining only Ascl1 and another transcription factor called Mef2c.

In further experiments they found evidence that Ascl1 on its own activates both neuron and cardiomyocyte genes, but it shifts away from the pro-neuron role when accompanied by Mef2c. In synergy with Mef2c, Ascl1 switches on a broad set of cardiomyocyte genes.

"Ascl1 and Mef2c work together to exert pro-cardiomyocyte effects that neither factor alone exerts, making for a potent reprogramming cocktail," Qian said.

The results show that the major transcription factors used in direct cellular reprogramming aren't necessarily exclusive to one targeted cell type.

Perhaps more importantly, they represent another step on the path towards future cell-reprogramming therapies for major disorders. Qian says that she and her team hope to make a two-in-one synthetic protein that contains the effective bits of both Ascl1 and Mef2c, and could be injected into failing hearts to mend them.

"Cross-lineage Potential of Ascl1 Uncovered by Comparing Diverse Reprogramming Regulatomes" was co-authored by Haofei Wang, Benjamin Keepers, Yunzhe Qian, Yifang Xie, Marazzano Colon, Jiandong Liu, and Li Qian.

Funding was provided by the American Heart Association and the National Institutes of Health (T32HL069768, F30HL154659, R35HL155656, R01HL139976, R01HL139880).


Story Source:

Materials provided by University of North Carolina Health CareNote: Content may be edited for style and length.


Journal Reference:

  1. Haofei Wang, Benjamin Keepers, Yunzhe Qian, Yifang Xie, Marazzano Colon, Jiandong Liu, Li Qian. Cross-lineage potential of Ascl1 uncovered by comparing diverse reprogramming regulatomesCell Stem Cell, 2022; 29 (10): 1491 DOI: 10.1016/j.stem.2022.09.006

World's first stem cell treatment for spina bifida delivered during fetal surgery

 Three babies have been born after receiving the world's first spina bifida treatment combining surgery with stem cells. This was made possible by a landmark clinical trial at UC Davis Health.

The one-of-a-kind treatment, delivered while a fetus is still developing in the mother's womb, could improve outcomes for children with this birth defect.

Launched in the spring of 2021, the clinical trial is known formally as the "CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele." Thirty-five patients will be treated in total.

The three babies from the trial that have been born so far will be monitored by the research team until 30 months of age to fully assess the procedure's safety and effectiveness.

The first phase of the trial is funded by a $9 million state grant from the state's stem cell agency, the California Institute for Regenerative Medicine (CIRM).

"This clinical trial could enhance the quality of life for so many patients to come," said Emily, the first clinical trial participant who traveled from Austin, Tex. to participate. Her daughter Robbie was born last October. "We didn't know about spina bifida until the diagnosis. We are so thankful that we got to be a part of this. We are giving our daughter the very best chance at a bright future."

Spina bifida, also known as myelomeningocele, occurs when spinal tissue fails to fuse properly during the early stages of pregnancy. The birth defect can lead to a range of lifelong cognitive, mobility, urinary and bowel disabilities. It affects 1,500 to 2,000 children in the U.S. every year. It is often diagnosed through ultrasound.

While surgery performed after birth can help reduce some of the effects, surgery before birth can prevent or lessen the severity of the fetus's spinal damage, which worsens over the course of pregnancy.

"I've been working toward this day for almost 25 years now," said Diana Farmer, the world's first woman fetal surgeon, professor and chair of surgery at UC Davis Health and principal investigator on the study.

The path to a future cure

As a leader of the Management of Myelomeningocele Study (MOMS) clinical trial in the early 2000s, Farmer had previously helped to prove that fetal surgery reduced neurological deficits from spina bifida. Many children in that study showed improvement but still required wheelchairs or leg braces.

Farmer recruited bioengineer Aijun Wang specifically to help take that work to the next level. Together, they launched the UC Davis Health Surgical Bioengineering Laboratory to find ways to use stem cells and bioengineering to advance surgical effectiveness and improve outcomes. Farmer also launched the UC Davis Fetal Care and Treatment Center with fetal surgeon Shinjiro Hirose and the UC Davis Children's Surgery Center several years ago.

Farmer, Wang and their research team have been working on their novel approach using stem cells in fetal surgery for more than 10 years. Over that time, animal modeling has shown it is capable of preventing the paralysis associated with spina bifida.

It's believed that the stem cells work to repair and restore damaged spinal tissue, beyond what surgery can accomplish alone.

Preliminary work by Farmer and Wang proved that prenatal surgery combined with human placenta-derived mesenchymal stromal cells, held in place with a biomaterial scaffold to form a "patch," helped lambs with spina bifida walk without noticeable disability.

"When the baby sheep who received stem cells were born, they were able to stand at birth and they were able to run around almost normally. It was amazing," Wang said.

When the team refined their surgery and stem cells technique for canines, the treatment also improved the mobility of dogs with naturally occurring spina bifida.

A pair of English bulldogs named Darla and Spanky were the world's first dogs to be successfully treated with surgery and stem cells. Spina bifida, a common birth defect in this breed, frequently leaves them with little function in their hindquarters.

By their post-surgery re-check at 4 months old, Darla and Spanky were able to walk, run and play.

The world's first human trial

When Emily and her husband Harry learned that they would be first-time parents, they never expected any pregnancy complications. But the day that Emily learned that her developing child had spina bifida was also the day she first heard about the CuRe trial.

For Emily, it was a lifeline that they couldn't refuse.

Participating in the trial would mean that she would need to temporarily move to Sacramento for the fetal surgery and then for weekly follow-up visits during her pregnancy.

After screenings, MRI scans and interviews, Emily received the life-changing news that she was accepted into the trial. Her fetal surgery was scheduled for July 12, 2021, at 25 weeks and five days gestation.

Farmer and Wang's team manufactures clinical grade stem cells -- mesenchymal stem cells -- from placental tissue in the UC Davis Health's CIRM-funded Institute for Regenerative Cures. The cells are known to be among the most promising type of cells in regenerative medicine.

The lab is a Good Manufacturing Practice (GMP) Laboratory for safe use in humans. It is here that they made the stem cell patch for Emily's fetal surgery.

"It's a four-day process to make the stem cell patch," said Priya Kumar, the scientist at the Center for Surgical Bioengineering in the Department of Surgery, who leads the team that creates the stem cell patches and delivers them to the operating room. "The time we pull out the cells, the time we seed on the scaffold, and the time we deliver, is all critical."

A first in medical history

During Emily's historic procedure, a 40-person operating and cell preparation team did the careful dance that they had been long preparing for.

After Emily was placed under general anesthetic, a small opening was made in her uterus and they floated the fetus up to that incision point so they could expose its spine and the spina bifida defect. The surgeons used a microscope to carefully begin the repair.

Then the moment of truth: The stem cell patch was placed directly over the exposed spinal cord of the fetus. The fetal surgeons then closed the incision to allow the tissue to regenerate.

"The placement of the stem cell patch went off without a hitch. Mother and fetus did great!" Farmer said.

The team declared the first-of-its-kind surgery a success.

Delivery day

On Sept. 20, 2021, at 35 weeks and five days gestation, Robbie was born at 5 pounds, 10 ounces, 19 inches long via C-section.

"One of my first fears was that I wouldn't be able to see her, but they brought her over to me. I got to see her toes wiggle for the first time. It was so reassuring and a little bit out of this world," Emily said.

For Farmer, this day is what she had long hoped for, and it came with surprises. If Robbie had remained untreated, she was expected to be born with leg paralysis.

"It was very clear the minute she was born that she was kicking her legs and I remember very clearly saying, 'Oh my God, I think she's wiggling her toes!'" said Farmer, who noted that the observation was not an official confirmation, but it was promising. "It was amazing. We kept saying, 'Am I seeing that? Is that real?'"

Both mom and baby are at home and in good health. Robbie just celebrated her first birthday.

The CuRe team is cautious about drawing conclusions and says a lot is still to be learned during this safety phase of the trial. The team will continue to monitor Robbie and the other babies in the trial until they are 6 years old, with a key checkup happening at 30 months to see if they are walking and potty training.

"This experience has been larger than life and has exceeded every expectation. I hope this trial will enhance the quality of life for so many patients to come," Emily said. "We are honored to be part of history in the making."

Video: https://youtu.be/TGvHRqsopQo


Story Source:

Materials provided by University of California - Davis Health. Original written by Tricia Tomiyoshi. Note: Content may be edited for style and length.


https://www.sciencedaily.com/releases/2022/10/221006164820.htm

HHS Probed EcoHealth Over Alleged 'Major Fraud' Against US, Emails Show

 by Eva Fu via The Epoch Times (emphasis ours),

The Department of Health and Human Services’ watchdog had briefly probed EcoHealth Alliance, a New York non-profit that has collaborated with the Wuhan Institute of Virology, over alleged “major fraud against the United States,” newly released emails show.

The four-month-long probe, which opened in September 2020, centered around an allegation that “the COVID 19 virus was generated in the China [sic] with the assistance of an NIH [National Institutes of Health] Grant.”

The Office of Investigations of the Department of Health and Human Services (HHS) revealed the investigation in an email with the subject line “Grant Information Review [Redacted]” and dated Dec. 3, 2020, according to redacted copies of the documents obtained by the U.S. Right to Know, a public health advocacy group.

A tip from an outside source had prompted the office to open the case, a special agent explained in an attached memorandum to Ashley Sanders, an investigative officer at the NIH. Another correspondence issued in January 2021 confirmed that EcoHealth Alliance, which for years worked with a high-level Wuhan lab to conduct risky bat coronavirus research, was one of the targets. The name of the other party was redacted.

Such allegations, if true, would constitute a violation of the U.S. federal law, Title 18 of U.S. Code §1031, “major fraud against the United States,” according to the memorandum. Under this law, anyone who tried “to obtain money or property by means of false or fraudulent pretenses, representations, or promises” in grant, contract, subcontract, or other forms of federal assistance valuing $1 million or more could receive a $1 million fine or up to 10 years in prison, or both.

The office referred the case to its special investigative branch on Sept. 9, 2020, and agents reviewed the allegation and met with NIH representatives on the same day, the memorandum said. Details of the meeting and a description of the subsequent step taken by the agents on Sept. 29 were redacted.

The investigators closed the probe on Jan. 11, 2021. In a letter informing Sanders of the decision, a special agent said they were asked to support efforts “regarding a high-profile situation that involved [redacted] HHS Grantee that had ties to the COVID 19 virus.” The reason for such a move was entirely redacted except for the word “developments,” although the agent noted the agency “reserves the right to reopen the investigation if any new, relevant information is discovered relating to this incident.”

The revelation of the existence of the investigation came as NIH, which is overseen by HHS, approved millions in new grants to EcoHealth for studies in Asia into novel viruses that could infect humans and cause an outbreak.

The NIH in August terminated funding for EcoHealth’s Chinese partner, the Wuhan Institute of Virology (WIV), citing failed efforts to gain lab entries and other records relating to the facility’s experiments subject of the grant.

https://www.zerohedge.com/political/hhs-probed-ecohealth-over-alleged-major-fraud-against-us-emails-show

California Begins Fighting Inflation With Stimulus Checks This Week

 Fighting fire with fire...

In true "government exercises every wrong solution before finally begrudgingly arriving at the correct one" fashion, this is the week that California residents can finally expect to get their "Middle Class Tax Refund" - the name the state has given to its stimulus checks it is distributing to residents in order to help fight inflation.

23 million taxpaying California residents are eligible to receive the payments, ABC reported this week. The payments are slated to start going out Thursday and Friday and are technically "tax refunds". 

The checks, which can be as much as $1,050 and are the brain child of Governor Gavin Newsom - will be distributed in amounts determined by people's annual income, mixed with the amount of dependents they have. The California Globe broke down the details this week:

...those making up to $75,000 a year, or joint filers who make up to $150,000, will get $350 each. Those who make up to $125,000 a year, or joint filers making up to $250,000, will get $250 each. Those who make up to $250,000 a year, or $500,000 filing jointly, get $200 each. One dependent may also be added at each tier for the same amount as each filer, meaning Californians could see as much $1,050, $750, or $600 coming in per household depending on tier level.

The checks were debated over the course of the spring and summer and first started under the guise of gas relief checks. But California legislators weren't happy with that idea and instead pushed for the idea of a flat rebate program for all residents making under a certain income level.

Sounds a lot like universal basic income, doesn't it?

People who filed their taxes online will receive a relief check direct deposit, the report says, while those who filed a paper tax return in 2020 will get their funds on a debit card. 90% of direct deposits are expected to hit accounts in October, the report says. Debit cards will be mailed between now and mid-December, the same report says. Some will receive their cards by mid-January 2023. 

Some have speculated that the timing of the stimulus conveniently lines up with mid-term elections, approaching next month. Former lobbyist Harry Schultz commented: “It’s not nearly as drastic this year since he has such a commanding lead, but this does help Newsom right before the election. Free money going out before an election, right during the beginning of a recession with consumer prices still being high? Yeah, that buys goodwill.”

He continued: “Honestly, it’s a good bet hedger. Just in case something catastrophic comes out that hurts Newsom, he still has some cards to play, and this is one of them. It has been odd timing both times to say the least, and if I were [Newsom Gubernatorial opponent Senator Brian] Dahle, I’d bring this up during the debate, even just a passing mention of it being ‘coincidental.'”

Meanwhile, as Insider notes, 73% of respondents said government spending was a "major cause" of higher prices when polled by a right-leaning advocacy group earlier this year. 

https://www.zerohedge.com/markets/california-begins-fighting-inflation-stimulus-checks-week

Biotech Hasn't Been This Blazing Hot Since Early 2021 — Here Are The Top 5

 Biotech stocks have been on a roller-coaster ride, but continue to outperform the broader market.

In 2021, the industry was thrust into the pandemic limelight as Pfizer (PFE) and its partner BioNTech (BNTX), Moderna (MRNA) and Johnson & Johnson (JNJ) launched a trio of Covid vaccines. But as society learned to live with Covid — and other concerns around the economy, inflation and politics took center stage — interest in biotech fell by the wayside. Shares of Investor's Business Daily's biotech industry group largely declined for 16 months.

Until now.

After hitting a six-year low in mid-June, biotech stocks seem to have regained their footing. Shares rose 40% from that point through mid-August, before tapering slightly down. But the slight decline comes amid a steeper dive for the broader market. The group is ranked fifth out of 197 industry groups tracked by Investor's Business Daily. Meanwhile, the pharma group ranks No. 164.

"The sector has started to show signs of a rebound, and we believe several key data events in the coming months as well as any pickup in (mergers and acquisitions), will likely be pivotal in determining whether investor risk appetite will increase and help sustain the recovery," RBC Capital Markets analyst Brian Abrahams said in a recent report.

But it's key to watch specific measures when examining stocks. In terms of fundamental and technical measures as well as 12-month performance, the best biotech stocks today are:

  • Catalyst Pharmaceuticals (CPRX)
  • Vertex Pharmaceuticals (VRTX)
  • Genmab (GMAB)
  • Neurocrine Biosciences (NBIX)
  • Astria Therapeutics (ATXS)

The No. 1 Biotech Stock

Catalyst is the No. 1 biotech stock, leading an industry group of more than 800 companies.

Its only product is Firdapse, a treatment for Lambert-Eaton myasthenic syndrome, or LEMS. LEMS is a rare autoimmune condition that saps muscle strength and often occurs in lung cancer patients.

Though sales are growing, the company is looking to acquire a second or third product — either through an outright buyout or licensing deal.

Now, the biotech stock is extended above a cup base with a buy point at 8.74, MarketSmith.com shows.

Catalyst shares are also on the Tech Leaders list.

Bullishly, CPRX shares have a best-possible Composite Rating of 99, according to IBD Digital. This puts the biotech stock in the top 1% of all stocks in terms of fundamental and technical measures. Its 12-month performance, measured by the Relative Strength Rating, is also in the top 1% of all stocks.

Moving Beyond Cystic Fibrosis

Vertex is one of the biggest biotech stocks in terms of market cap. It ranks fourth behind Amgen (AMGN), Gilead Sciences (GILD) and Regeneron Pharmaceuticals (REGN).

The company is the de facto leader of the cystic fibrosis drug market. Second-quarter sales — dominated by its triple regimen Trikafta — jumped 22% to almost $2.2 billion.

But it's now expanding into other efforts. Vertex is partnered with Crispr Therapeutics (CRSP) on a gene-editing approach to a pair of blood diseases. Further, Vertex recently announced its $320 million plan to buy its partner in diabetes treatment, privately held ViaCyte. The companies are testing a cell replacement drug in type 1 diabetes.

Beyond that, Vertex is testing treatments for liver and kidney diseases, Duchenne muscular dystrophy and pain.

The biotech stock has a perfect Composite Rating and a Relative Strength Rating of 96. Shares are now above their 50-day moving average.

The company is also a Tech Leader.

Cancer Treatments Are Key

Genmab sells a small handful of treatments for cancer. The company partners with big biopharma names, including AbbVie (ABBV), Johnson & Johnson (JNJ), Horizon Therapeutics (HZNP) and Seagen (SGEN).

In the second quarter, adjusted earnings and sales increased bullishly to 40 cents per share and $440 million, respectively. Both measures also easily topped forecasts.

Now, Genmab on expanding its suite of medicines to new solid tumors and blood cancers.

Shares have a nearly perfect Composite Rating of 98, but a middling Relative Strength Rating of 69. Genmab is also a Tech Leader.

Gaining Pipeline Recognition

Biotech stock investors are watching catalysts galore for Neurocrine Bio in the second half of 2022 and next year. The company sells a movement disorder treatment called Ingrezza. Ingrezza brought in $350 million in net sales in the second quarter, growing by double digits and beating forecasts.

Neurocrine also raised its Ingrezza sales expectations for the year to $1.35 billion to $1.4 billion.

Meanwhile, the company has a number of drugs in the works for schizophrenia, depression and an adrenal disorder — to name just a few.

The biotech stock's relative strength line is at a 52-week high. Shares also have a promising Composite Rating of 97 and a Relative Strength Rating of 93. Neurocrine stock briefly topped a buy point at 109.36 out of a flat base this month.

Plus, NBIX shares land on the Tech Leaders list.

Biotech Stock: Angling For Hereditary Angioedema

Astria Therapeutics is working in the increasingly competitive area of hereditary angioedema. It's testing a monoclonal antibody that it hopes will protect against swelling attacks. The company expects to have Phase 1 test results before year-end.

As others testing hereditary angioedema treatments have faced struggles, Astria stock has soared. Early this week, Kalvista Pharmaceuticals (KALV) scrapped its hereditary angioedema drug due to safety concerns. While Kalvista stock plummeted, Astria shares climbed almost 17%.

The segment is heating up with Intellia Therapeutics (NTLA) testing a gene-editing approach to the genetic disease. Biocryst Pharmaceuticals (BCRX) and Takeda Pharmaceutical (TAK) with Ionis Pharmaceuticals (IONS) make treatments for the condition.

https://www.investors.com/news/technology/biotech-stocks-the-top-5-to-watch-amid-a-blazing-hot-run/

Advocates tell top Canadian court US is not safe for asylum-seekers

 Lawyers advocating for refugee safety argued before the Canadian Supreme Court on Thursday that the U.S. is unsafe for asylum-seekers, challenging the constitutionality of the Safe Third Country Agreement.

The 2004 treaty allows the neighboring countries to share responsibility for migrants seeking asylum, obligating refugees to remain in whichever of the two nations they first enter after fleeing their homelands.

The pact is based on the assumption that the Canadian and American governments operate off of similar constitutional values of “life, liberty and security of the person” and remains in operation as long as both fulfill their obligations under international law.

“The underlying issue is whether or not the obligation to provide effective protection and to ensure effective protection is being respected by the country to which Canada is transferring refugee claimants,” said attorney Andrew J. Brouwer, advocating for refugees, according to The Washington Post.

He continued: “Our submission on the evidence is that it’s not.”

Brouwer represents three families or individuals who attempted to flee the U.S. for Canada, as well as multiple refugee advocacy organizations.

The Post reports that the appellants included an Ethiopian woman who feared that her Oromo ethnicity would subject her to persecution, a Salvadoran woman and her daughters who experienced gender-based violence and a Syrian family who fled to Canada after former President Trump issued an executive order preventing citizens of seven predominately Muslim countries, including their homeland, from entering the U.S.

The asylum-seekers and advocacy groups argue that the Safe Third Country Agreement opens Canada and the U.S. up to potential violations of the international human rights principle of non-refoulement, which protects refugees from being forcibly returned to the country that they have fled, because of the possibility that the American government would reject their settlement in the country.

Canadian government lawyer Marianne Zoric countered this idea, according to the Post, arguing that the “necessary elements of what is required for a safe third country are met in terms of international law” when it comes to the U.S.-Canada agreement.

https://thehill.com/policy/international/3678274-advocates-tell-top-canadian-court-us-is-not-safe-for-asylum-seekers/