Gene therapy cures patients with hemophilia B

Story at a glance


  • Hemophilia B occurs when patients lack sufficient levels of the blood protein factor IX.

  • Some current treatments for the condition involve repeated infusions of the protein.

  • But new research shows a one-time gene therapy could offer long-term efficacy for patients.

Hemophilia B is a rare condition that prevents patients’ blood from sufficiently clotting, heightening the risks of prolonged bleeding. 

However, new trial results published today led researchers to claim the majority of adults with the disorder could be cured within the next three years.

This is thanks to FLT180a (verbrinacogene setparvovec), a gene therapy that functions to normalize levels of factor IX, a blood protein that is insufficient in patients with the condition. Findings of the multicenter trial, which included 10 patients with severe or moderately severe hemophilia B, were published in The New England Journal of Medicine

After 26 weeks of receiving different doses of FLT180a, patients were enrolled in a longer follow-up study. After 27 months, all patients but one exhibited sustained levels of factor IX.


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Around 10 percent of adverse events reported throughout the study period were associated with the treatment while around a quarter were due to immunosuppression, resulting from use of glucocorticoids with or without tacrolimus – a substance used to help the body prevent rejection of a transplanted organ. 

Questions about FLT180a remain, including how long its benefits will last in patients, with some estimates projecting it could remain effective for at least a decade. 

Gene therapies have been making waves in the medical field due to their lasting efficacy, while current trials are underway to study the treatments’ effects in a variety of diseases, ranging from cancer to glaucoma. 

These therapies work by introducing a new or replacement gene to the patient through a viral vector administered via an infusion. Despite the seemingly long-term efficacy seen with these therapies, they are often expensive and the majority must be delivered in an in-patient setting. But for some, including FLT180a, only a single treatment is needed for patients to reap the benefits. 

Only a handful of gene therapies are approved in the United States, including a treatment for spinal muscular atrophy (SMA) and elapsed acute lymphoblastic leukemia. Zolgensma, the treatment approved for SMA, currently costs 2.1 million U.S. dollars for a one-time infusion, making it the most expensive drug in the country as of 2021. 

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Russia calls for US to respect its laws in Griner case

A Russian official has said the U.S. must respect its laws with regards to WNBA star Brittney Griner, who has been detained in Russia.

Moscow’s foreign ministry spokesperson Maria Zakharova said on Thursday that  U.S. officials disrespected  Russian law by characterizing Griner as being wrongfully detained, according to a report by ABC News.

Zakharova also said U.S. laws on medical and recreational cannabis use have no bearing on what happens in Russia.

“If a U.S. citizen was taken in connection with the fact that she was smuggling drugs, and she does not deny this, then this should be commensurate with our Russian, local laws, and not with those adopted in San Francisco, New York and Washington,” Zakharova said.

“You understand, if drugs are legalized in the United States, in a number of states, and this is done for a long time, and now the whole country will become drug-addicted, this does not mean that all other countries are following the same path,” Zakharova added. 

Griner, a seven-time WNBA All-Star and Olympic gold medalist, has been detained in Russia since February on charges of possessing vape cartridges with cannabis oil in her luggage at a Moscow airport. 

Griner, who plead guilty to drug possession charges earlier this month, could face up to 10 years in prison if convicted. The State Department in May classified Griner as “wrongfully detained” in Russia. 

Zakharova’s remarks come after Deputy Foreign Minister Sergei Ryabkov said earlier this month that the U.S. attempts to “foment hype and make noise” around Griner’s case isn’t going to resolve any issues. 

The Biden administration has said that Griner’s case is a top priority for them after receiving criticism for a lack of action to achieve Griner’s release to U.S. soil and minimal effort to speak with Griner’s family on the matter. 

Golden State Warriors star Stephen Curry, along with fellow WNBA stars Nneka Ogwumike and Skylar Diggins-Smith, Griner’s Pheonix Mercury teammate, called for the Griner’s release from detainment during the 2022 ESPY awards show on Wednesday.

“They’re a lot of people working hard right now to secure Brittney’s release, but as we hope for the best, we urge the entire global sports community to continue to stay energized on her behalf,” Curry, who served as the shows host, said during the segment.

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Bannon will not present a defense case at trial

Steve Bannon’s lawyers will not call any witnesses or introduce any new evidence in his criminal contempt trial after prosecutors spent less than two days laying out their case against the former White House strategist.

David Schoen, one of the attorneys representing Bannon, told a federal judge Thursday morning they will not mount a defense in the trial over whether Bannon willfully defied a subpoena from the House Jan. 6 Select Committee.

The move means the jury in the case, which was impaneled on Tuesday, will not hear testimony from Bannon himself. The defense team had previously floated possibly having him take the stand.

Both sides will be able to make closing arguments to a jury before they begin deliberating on a verdict.

Prosecutors rested their case Wednesday afternoon, after calling just two witnesses.

While Bannon’s lawyers don’t intend to call any of their own witnesses, they have been aggressive in cross-examining the prosecution’s.

Evan Corcoran, another member of the defense team, tried to paint the 2021 subpoena’s deadlines as flexible, introduced Bannon’s belated offer earlier this month to testify before the committee and attempted to paint the decision to hold Bannon in contempt and to prosecute him as politically motivated.

U.S. District Judge Carl Nichols has prohibited the defense team from arguing that Bannon was shielded from complying with the subpoena because of executive privilege or from calling the jury’s attention to the potential political motivations of the Jan. 6 committee.

He issued a warning Wednesday that politics would not overcome the facts in the case.

“I do not intend for this to become a political case, a political circus, a forum for partisan politics,” Nichols, a Trump appointee, said Wednesday morning.

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Amazon acquiring One Medical for nearly $4 billion

Amazon is buying the primary health care provider One Medical for roughly $3.9 billion, the companies announced Thursday.

The deal expands the e-commerce giant’s footprint in the healthcare space, an area that Amazon said is ripe for “reinvention.”

“Booking an appointment, waiting weeks or even months to be seen, taking time off work, driving to a clinic, finding a parking spot, waiting in the waiting room then the exam room for what is too often a rushed few minutes with a doctor, then making another trip to a pharmacy – we see lots of opportunity to both improve the quality of the experience and give people back valuable time in their days,” Neil Lindsay, the senior vice president of Amazon Health Services, said in a statement.

The purchase, an all cash transaction at $18 a share, is subject to approval by regulators and One Medical’s shareholders.

One Medical CEO Amir Dan Rubin will remain atop the company if the deal is completed.

The primacy care provider, which went public in 2020, operates 188 medical offices in 25 markets, according to its latest quarterly results.

One Medical also offers a variety of telemedicine services.

This is not Amazon’s first purchase in the health care space. It acquired PillPack for $750 million in 2018 before launching its own online pharmacy.

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No, half of small businesses aren’t ‘in jeopardy of closing’ by fall — not even close

As someone who writes about small business, I receive lots of ridiculous surveys from companies and their public relations (PR) firms looking for a headline. But one that hit my inbox recently must be the most ridiculous of them all. It’s a survey sponsored by Alignable, a legitimate and popular online network for small businesses.

Alignable hired an outside firm to survey 4,392 small business owners between mid-June and mid-July and came away with this eye-grabbing headline: “47 Percent Of SMBs Risk Closing By Fall.”

What could possibly be the reason for such a devastating prediction? The company’s head of PR says it’s the consequence of “record-breaking inflation, higher-than-normal gas prices, rent hikes, labor issues, a still-broken supply chain, reports of reduced consumer spending, elevating interest rates, recessionary fears, and the fact that several businesses have not recouped losses from 2020-2021 yet.”

I run a firm that serves about 600 small and mid-sized clients. My clients are also facing these challenges. But none of them – at least to my knowledge – would say they’re going out of business because of them. And yet almost half of those in the survey apparently think they will. That seems very, very dubious.

Dubious because there are more than 30 million small businesses in this country. If 47 percent go out of business, that means nearly 15 million shuttered small businesses, greatly outpacing the 1.8 million that reportedly closed during the Great Recession.

In 2021, a year after a global pandemic that shut down all of the world’s major economies for periods of time, bankruptcy filings actually dropped compared to 2020, according to data released by the Administrative Office of the U.S. Courts.

For perspective, during the first 11 months of 1980 – a period at the height of a historic inflation/interest spiral – the New York Times reported that 10,727 businesses filed bankruptcy petitions, which was 52 percent more than the number filed in the same period in 1979, yet still only a small fraction of the total businesses in operation around the U.S.

Regarding “record-breaking inflation, higher-than-normal gas prices, rent hikes” and a “still-broken supply” chain — all these issues are real.

But gas prices – as well as many of the core material costs from industrial chemicals, packaging supplies, domestic shipping, iron and steel and lumber – now appear, after a period of spiking, to be plateauing. West Coast ports are returning to normal operations. Inventories are building, not declining. Inflation will likely remain elevated over the next year. But few are shutting their doors because of it. Most businesses are doing what businesses have done since the time of Cleopatra: raising prices, finding alternative supply, switching up products, watching their overhead, taking care of their customers.

Labor shortages and financing are a problem, but small businesses have been dealing with these challenges since before the pandemic. Even the National Federation of Independent Businesses acknowledges that half of small businesses still have job openings, and data from major banks like JPMorgan Chase, Wells Fargo and Citigroup show that Main Street is “humming along,” credit card spending “still looks healthy” and that “while banks are putting aside more money to cover bad loans, they aren’t seeing significant problems yet.”

Fifteen million small businesses aren’t going to close by fall — not even close. It’s already mid-summer. Restaurants are open and busy. Stores have traffic. Businesses are shipping products, building homes, performing services. Some more than others. Some less than others.

The survey makes small businesses look incompetent and inept. It gives the perception that small business owners are whiners who, unable to solve problems, simply quit. It makes it harder for small business owners to recruit employees and build credibility with prospective customers and partners. And it undermines their investors’ and lenders’ confidence in future projects.

And yet, as ridiculous as this study is – and, let’s be honest, because it’s so ridiculous – it gets covered in the media. But its main effect is not to inform but needlessly to create fear and uncertainly — two things we could all use a little less of right now.  

Gene Marks is founder of The Marks Group, a small-business consulting firm. He frequently appears on CNBC, Fox Business and MSNBC.

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Oxford shooter parents seek to have cases against them thrown out

The parents of an accused teen school shooter in Michigan filed a motion on Monday to dismiss a case against them, arguing they are not responsible for the actions of their son.

Defense attorneys for James and Jennifer Crumbley filed the motion in Michigan’s Court of Appeals, claiming there are no legal grounds to prosecute them on involuntary manslaughter charges and alleging the state was trying to make an example out of the parents for an issue that belongs in the legislature, according to the Detroit Free Press.

“Certainly, if the prosecution could directly link Mr. or Mrs. Crumbley to the mass shooting, they would be prosecuted for first-degree murder as if they had directly committed the offense,” the motion reads, according to the news outlet, which reviewed the filing. “However, because the prosecution cannot support such a claim, they are left attempting to fit a square peg into a round hole.”

Ethan Crumbley, 16, shot and killed four people and injured seven others after he opened fire inside Oxford High School on Nov. 30. Ethan Crumbley faces first-degree murder and terrorism charges. He has pleaded not guilty.

James and Jennifer Crumbley were charged with involuntary manslaughter after prosecutors alleged they bought a handgun for their son and ignored warning signs that he had violent tendencies. They have both pleaded not guilty.

The parents are set for a trial next month before Oakland County Circuit Judge Cheryl Matthews, who has allowed the case to proceed and argued there is enough evidence for a trial.

After Mathews denied a request from the parents to quash the charges, the defense filed a motion in the Michigan Court of Appeals to dismiss, according to the Detroit Free Press.

The defense has argued the gun was secured in the home and they had no idea their son was going to use it.

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Jan. 6 hearing to show Trump spent day largely watching violence unfold on TV

President Trump was primarily sitting in the White House dining room watching T.V. while the deadly attack unfolded at the Capitol on Jan. 6, aides told the committee investigating the riot.

The clip, shared by Rep. Adam Kinzinger (R-Ill.), who will help lead Thursday’s primetime hearing, shows numerous aides indicating Trump spent the duration of the attack away from his desk watching the violence unfold.

“To the best of my recollection he was always in the dining room,” Kayleigh McEnany, Trump’s former White House press secretary, says in the clip.

“I think everybody was watching the T.V,” Keith Kellogg, national security advisor to former Vice President Mike Pence, told the committee.

“It’s my understanding he was watching television,” said Molly Michael, an executive assistant to Trump. 

The clip was released ahead of a hearing in which the committee will explore the 187 minutes between Trump finishing his speech at the White House Ellipse until shortly after 4 p.m., when he released a video asking the rioters to go home.

The committee plans to show Trump was aware the situation was escalating to violence, but chose to do little to intervene.

When asked if the violence playing out was visible on the T.V. screen, Pat Cipollone, Trump’s White House counsel, simply answered, “Yes.”

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Groups demand DOJ explain position on whether Trump could be prosecuted

A coalition of progressive groups asked the Justice Department (DOJ) on Thursday to explain its internal policies on the question of whether presidents can be charged with a crime.

Twelve advocacy and watchdog organizations sent a letter to Attorney General Merrick Garland calling on him to clarify the DOJ’s position as the House committee investigating the Jan. 6 attack builds its case that Trump broke the law to undermine the 2020 election results.

The groups said they are concerned that the department’s longstanding legal positions may be constraining federal law enforcement from seriously considering the possibility of charging Trump with a crime despite growing evidence that could support prosecuting him under the same criminal statutes the DOJ has used against hundreds of Jan. 6 rioters.

“If the Justice Department believes that presidents of the United States are exempt from 18 U.S.C. § 1512(c)(2) or from any other criminal law based on a ‘clear statement rule,’ it must tell the public and Congress, to give Congress the opportunity to close this indefensible loophole,” the letter reads, citing the criminal statute for obstruction of an official proceeding.

“If our fears are groundless, and the Justice Department believes that former presidents are subject to the same criminal laws as every other citizen, it is entirely appropriate to reassure the public of that fact.”

Groups that signed the letter included: Project on Government Oversight, Citizens for Responsibility and Ethics in Washington, the Constitutional Accountability Center, Public Citizen and the Revolving Door Project.

A spokesman for the Justice Department did not immediately respond when asked to comment on the letter.

Despite some signs that the DOJ’s Jan. 6 investigation has moved closer to Trump, it’s unclear whether the department’s leadership is seriously considering taking the unprecedented step of charging a former president with a crime.

The groups behind Thursday’s letter said they’re concerned binding legal memos from the DOJ’s Office of Legal Counsel (OLC) going back decades might be seen as roadblocks for investigators.

They asked Garland to specifically explain how a guideline known as the “clear statement rule” might apply. The rule, articulated in several OLC memos since the 1990s, holds that the department must interpret criminal laws as “not applying to the President if such application would involve a possible conflict with the President’s constitutional prerogatives.”

The OLC, which issues legal opinions that are binding on the entire executive branch, often shields its memos from the public. Government transparency advocates say the opaque office is allowed to essentially create secret laws with little oversight.

In 2019, then-Special Counsel Robert Mueller said his office understood that it was bound by OLC opinions holding that a sitting president could not be charged with a crime.

The president and the attorney general have the authority to override OLC opinions, but it remains largely unclear which of the office’s memos remain in effect today, or whether the department’s leadership sees any of them as an impediment to investigating or prosecuting Trump.

“To the extent that nonpublic Justice Department memoranda related to this subject exist, we strongly urge you to make such memoranda available to Congress and the public,” the coalition of progressive groups wrote Thursday.

Source: TEST FEED1

Don’t trust the news? You’re certainly not alone

This just in: There’s practically nobody left in the United States who still trusts the news media.  And if that’s an exaggeration, it’s not by much.

A new Gallup poll found that “Americans’ confidence in two facets of the news media — newspapers and television news — has fallen to all-time low points.”

Only 16 percent of us now say we have “a great deal” or “quite a lot” of confidence in newspapers, and even fewer Americans —11 percent — have that kind of confidence in television news. Of the 16 institutions Gallup tracks, only Congress ranks lower than TV news.  

If that doesn’t bother journalists, there’s a good chance that nothing will.

When I left CBS News, where I was a correspondent for 28 years, I wrote a book called “Bias,” about liberal bias in the news. It came out in 2001. Back then, journalists at least tried to hide their biases. Not anymore. On cable TV news shows, you’d have to be in a coma not to know where the talking heads stand politically — and that’s not only the “opinion” talking heads. The line separating straight news from opinion used to be a bright one; it’s a lot fuzzier these days because (and here’s the dirty little secret) bias is good for business.

Back to the poll: When it comes to partisan breakdowns, more Democrats trust the media than do Republicans. No surprise there. America’s journalists, by and large, are liberal — no one would seriously dispute that — so they tend to slant the news in a liberal direction, which pleases Democrats more than it does Republicans. But even Democrats have lost confidence in the news media — only 35 percent of them have “a great deal” or “quite a lot” of confidence in newspapers, according to Gallup, and only 20 percent have confidence in television news.

Rodney Dangerfield got more respect.

But there’s something we too often leave out of the discussion of bias in the news: the bias of the news consumer.

People complain about bias but a lot of them actually want biased news, no matter what they say. What they’re really against is the other side’s bias. People who watch Fox News typically have no problem with conservative bias, which they don’t even see as bias — and people who watch the liberal channels such as CNN or MSNBC, or read liberal newspapers, don’t complain about liberal bias, which they often see as reasonable, moderate, down-the-middle news.

And there’s even research that concluded that the more partisan the news consumer, the more likely he is to believe the news he’s getting is biased.  

In a 2018 piece, the New York Times reported, “The bias consumers bring with them distorts their rating of news content, new research shows, and those who are most distrustful of the news media tend to be the most biased readers. … Not surprisingly, those with more extreme political views tend to provide more biased ratings of news.”

The study by Gallup and the Knight Foundation also found that people identifying with the Republican Party were more likely to see bias in a story published in the New York Times when they knew the source of the story. But when they didn’t know the source, they found the news less biased. “Similarly, those identifying with the Democratic Party who read media perceived as right-leaning, like Fox News, rated it [less biased] when they did not know the source,” the Times reported.

So, if you’re a liberal, you think just about everything on Fox News is biased; if you’re a conservative, you think just about everything on CNN, MSNBC and in the New York Times is biased. And if that doesn’t bring you down, you’re not paying close enough attention.

Here’s the bottom line from Gallup: “Multiple Gallup measures of Americans’ views of the news media show a growing distrust. Last fall, Gallup found near-record low trust in the media to report the news fully, accurately and fairly, and few Americans rated newspapers and television reporters as highly ethical in Gallup’s annual honesty and ethics of professions poll in December. Although trust in the media in the U.S. has been scarce for many years, confidence ratings for newspapers and TV news have never been as low as they are now. Taken together, these data suggest that the media has a long way to go to win back the public’s confidence.”

Good luck with that. If history is any guide, journalists won’t care what the public thinks of them. There are more guys working the overnight shift at 7-Eleven selling cigarettes and beef jerky to insomniacs who have more introspection than a lot of journalists, who reflexively circle the wagons when they hear the word “bias.”

We all know that a democratic country such as ours needs a free press to survive. Russia may not need the kind of journalism that garners the confidence of its people. China may not. But we do. And for the long run, it’s not only a free press that we need to survive. We also need a fair press, news the public trusts. That’s something else, I suspect, that a lot of elite journalists won’t take seriously.

Bernard Goldberg is an Emmy and an Alfred I. duPont-Columbia University award-winning writer and journalist. He was a correspondent with HBO’s “Real Sports with Bryant Gumbel” for 22 years and previously worked as a reporter for CBS News and as an analyst for Fox News. He is the author of five books and publishes exclusive weekly columns, audio commentaries and Q&As on his Substack page. Follow him on Twitter @BernardGoldberg.

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Stephen Curry calls for release of Brittney Griner while hosting ESPYS

Stephen Curry took a moment during the ESPYs Wednesday night to call for the release of WNBA player Brittney Griner, who has been detained in Russia since February.

Curry, who hosted the sports awards show, was joined by WNBA players Nneka Ogwumike and Skylar Diggins-Smith in calling attention to Griner’s situation. The Phoenix Mercury center was detained at a Moscow airport after Russian police said they found vape canisters with cannabis oil in her luggage.

“BG is a WNBA champion,” Ogwumike said to the audience. “She’s an eight-time WNBA All Star. A national champion in college, an Olympic gold medalist. An athlete who has fans all over the world. A human being whose predicament commands our attention.”

“It’s been 153 nights now that BG has been wrongfully detained thousands of miles away from home, away from her family, away from her friends, away from her team,” Diggins-Smith said. “All throughout that time, we’ve kept her in our thoughts and in our hearts even though we know that ain’t nearly enough to bring her home, y’all.”

“They’re a lot of people working hard right now to secure Brittney’s release, but as we hope for the best, we urge the entire global sports community to continue to stay energized on her behalf,” Curry said.

“We cannot stop fighting for her. We cannot stop believing for her, and we will not stop hoping for the day when we can welcome her home safely,” Curry added.

The three athletes ended their remarks by saying together, “We are BG.”

Griner’s wife Cherelle was present at the ESPYs and applauded the calls to push for Griner’s release.

Soccer star Megan Rapinoe also spoke about Griner in her acceptance speech after winning the award for Best Play.

“I think honestly what we’ve witnessed tonight is the importance of sport and how much we can bring and how much we can get done in the world with our collective power,” Rapinoe said.

“Every time we say her name, it puts pressure on everyone — puts pressure on the administration, puts pressure on Russia. The most striking thing is that BG isn’t here. BG deserves to be free,” the soccer player added.

The comments bringing attention to Griner at the ESPYs come after the WNBA star pleaded guilty to drug possession charges in a Russian court earlier this month, which could result in a sentence of up to 10 years.

Vice President Harris told CBS’s Robert Costa in an interview just after Griner pleaded guilty that the Biden administration is making it a top priority to bring Griner home.

The administration has received criticism for what some have denounced as a lack of action to achieve Griner’s release and minimal effort to reach out to her family.

Source: TEST FEED1