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FDA official calls UniQure’s gene therapy a ‘failed’ treatment for Huntington’s disease

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FDA official calls UniQure’s gene therapy a ‘failed’ treatment for Huntington’s disease


Thomas Fuller | SOPA Images | Lightrocket | Getty Images

UniQure needs to run another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior U.S. Food and Drug Administration official said on a call with reporters Thursday.

The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled trial of its treatment, which is administered directly into the brain. UniQure has said that type of study isn’t ethical because it would require putting people under general anesthesia for hours, a characterization the official disputed.

“So what is really going on? UniQure is the latest company to make a failed therapy for Huntington’s patients,” the official said. “They likely acknowledge or understand at some deep level that their trial failed years ago, and instead of doing the right thing and running the correct clinical study, UniQure is performing a distorted or manipulated comparison in the mind of FDA.”

The comments mark the latest development in a messy public spat between UniQure and the FDA, and as the agency comes under fire for a number of recent drug approval application rejections, including some where companies have accused it of going back on previous guidance. FDA Commissioner Marty Makary in an interview with CNBC’s Becky Quick last week seemingly criticized UniQure’s gene therapy for Huntington’s disease. Makary didn’t name UniQure but described its treatment.

UniQure then accused the FDA of reversing its stance that the company’s clinical trial data would be sufficient to seek approval. UniQure’s study used an outside database to measure how patients with Huntington’s disease might decline without treatment, known as an external control. UniQure has said it wouldn’t be feasible to run a true randomized, double-blind placebo-controlled study, considered the gold standard, because it wouldn’t be ethical to make people undergo a sham hours-long brain surgery.

The FDA official said the agency “never agreed to accept this distorted comparison” and the FDA “never makes such assurances.” Instead, the “FDA will always say, ‘Well, we have to see the data when we get it.'”

UniQure didn’t immediately comment.

The company’s stock rose more than 10% on Thursday and has fallen 58% this year as of Thursday afternoon.



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Apple names new boss to replace Tim Cook after 15 years

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Apple names new boss to replace Tim Cook after 15 years



John Ternus will take over running the technology giant as Cook steps up to become executive chairman.



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SBP receives final $1bn from Saudi Arabia, bringing total deposit reaches $3bn – SUCH TV

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SBP receives final bn from Saudi Arabia, bringing total deposit reaches bn – SUCH TV



The State Bank of Pakistan (SBP) has received $1 billion from the Ministry of Finance of the Kingdom of Saudi Arabia, marking the second tranche of a $3 billion deposit agreed recently, the central bank said on Tuesday.

According to the statement issued by the central bank, the second tranche was received with a value date of April 20, 2026.

The first tranche of $2 billion had already been received on April 15, 2026, bringing the total inflows under the arrangement to $3 billion.

The development comes days after Prime Minister Shehbaz Sharif’s visit to Saudi Arabia, where he engaged in diplomatic efforts aimed at promoting regional peace.

During his visit, the premier met Crown Prince Mohammed bin Salman in Jeddah and expressed appreciation for the Kingdom’s continued support for Pakistan’s economic stability. He also conveyed solidarity with Saudi Arabia in light of recent regional developments.

Earlier on April 16, Finance Minister Muhammad Aurangzeb had announced that Saudi Arabia would provide $3 billion in additional financial support, with disbursement expected shortly.

He also noted that Riyadh had extended the tenure of its existing $5 billion deposit, removing the earlier annual rollover requirement.

The Saudi funding has strengthened Pakistan’s external position as it repaid $2 billion in debt to the United Arab Emirates (UAE).

The amount was kept with the central banks as a safe deposit.

Saudi Arabia has been a key financial partner for Pakistan, having provided support packages during previous economic challenges, including a $6 billion assistance programme in 2018 comprising deposits and oil facility arrangements.



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Oil prices dip, most stocks rise on lingering Iran peace hopes | The Express Tribune

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Oil prices dip, most stocks rise on lingering Iran peace hopes | The Express Tribune


Crude plunged on Friday after Tehran said it would allow ships to transit the Strait of Hormuz

A map showing the Strait of Hormuz, also known as Madiq Hurmuz, and 3D printed oil barrels are seen in this illustration taken March 26, 2026. PHOTO: REUTERS


HONG KONG:

With the end of a two-week ceasefire approaching, the White House said Vice President JD Vance was ready to return to Pakistan for fresh negotiations to end a conflict that has sent crude soaring and revived inflation fears.

However, the Islamic Republic’s position remained uncertain as it accused Washington of violating their fragile truce through its blockade of the country’s ports and seizure of a ship.

Crude plunged on Friday after Tehran said it would allow ships to transit the Strait of Hormuz, which had been effectively closed since the war began on February 28.

Read: US positive on Iran deal but talks still uncertain as ceasefire end nears

But the commodity rebounded on Monday as Iran closed the waterway again, citing the blockade and seizure.

Donald Trump has similarly accused Tehran of violating the ceasefire by harassing vessels in the Strait of Hormuz, the transit passage for about one-fifth of global oil.

The US president said the blockade would not be lifted until an agreement had been reached.

“THE BLOCKADE, which we will not take off until there is a ‘DEAL,’ is absolutely destroying Iran,” Trump said on social media. “They are losing $500 Million Dollars a day, an unsustainable number, even in the short run.”

He told PBS News that Iran was “supposed to be there” at the talks in Pakistan.

“We agreed to be there,” he said, warning that if the ceasefire expired “then lots of bombs start going off”.

He separately told Bloomberg News it was “highly unlikely” he would extend the truce.

Based on its start time, the truce theoretically expires overnight on Tuesday, Iran time, although in his comments to Bloomberg Trump said the end was Wednesday evening Washington time.

The Middle Eastern country’s parliament speaker, Mohammad Bagher Ghalibaf, said “Trump wants to turn this negotiating table into a surrender table or justify renewed hostilities, as he sees fit”.

“We do not accept negotiations under the shadow of threats, and in the last two weeks we have been preparing to show new cards on the battlefield,” he wrote on X.

Still, investors remained largely upbeat that the two sides will eventually come to a deal that will reopen the strategic strait.

US benchmark crude West Texas Intermediate rose more than 1%, while Brent was also higher.

Tech rally

Seoul led the equity market gains thanks to a resumption of the tech rally that had pushed the Kospi to multiple records before the war, while Tokyo and Taipei were also well up.

Hong Kong, Singapore and Manila also advanced, although Shanghai and Sydney fluctuated.

That came even after a down day on Wall Street, where the S&P 500 and Nasdaq Composite retreated from Friday’s record closes.

Asia had opened “with a gentle lean into risk as signs Iran may join talks with the US offer a pathway, however narrow, toward easing tensions ahead of the ceasefire deadline”, wrote SPI Asset Management’s Stephen Innes.

“Markets are pricing the possibility of progress rather than its certainty,” he said.

“Trump’s remark that a ceasefire extension is ‘highly unlikely’ if no deal is reached has effectively put a clock on the market.

“However, traders recognise the playbook. Hard deadlines and firm rhetoric often soften as negotiations evolve, but the presence of a timeline still sharpens positioning and raises the stakes around each headline.”

In company news, Japanese arms firms enjoyed healthy buying after Tokyo said on Tuesday it would ease decades-old export rules, paving the way for the sale of lethal weapons overseas.

The policy shift, which ends Tokyo’s self-imposed restraint on the sale of lethal arms, comes as it seeks to enter the international arms market, hoping to bolster national defence as well as boost economic growth.

Fujitsu climbed 2.4%, NEC added 3.7%, and Mitsubishi Electric was up 0.9%, while Mitsubishi Heavy gained 0.4%.

Key figures around 0230 GMT

West Texas Intermediate: DOWN 1.2% at $88.50 a barrel

Brent North Sea Crude: DOWN 0.4% at $95.12 a barrel

Tokyo – Nikkei 225: UP 1.3% at 59,596.10 (break)

Hong Kong – Hang Seng Index: UP 0.3% at 26,427.75

Shanghai – Composite: DOWN 0.2% at 4,073.82

Euro/dollar: DOWN at $1.1780 from $1.1786 on Monday

Pound/dollar: DOWN at $1.3525 from $1.3535

Dollar/yen: UP at 158.98 yen from 158.88 yen

Euro/pound: UP at 87.10 pence from 87.07 pence

New York – Dow Jones: FLAT at 49,442.56 (close)

London – FTSE 100: DOWN 0.6% at 10,609.08 (close)



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