Connect with us

Business

Rare disease biotech investment rises after PRV renewal

Published

on

Rare disease biotech investment rises after PRV renewal

Investment in rare disease biotechs has increased since the US Food and Drug Administration’s Rare Pediatric Disease Priority Review Voucher (PRV) programme was signed back into law in February, according to SynaptixBio, the only company licensed to commercialise a treatment for the rare, deadly disease H-ABC, though the United States remains dominant.

The programme, which grants tradable vouchers to developers of rare paediatric disease treatments, will remain in place until it is reviewed again in September 2029. Vouchers have recently been sold for between $150 million and $200 million, and because a sale does not require the seller to issue new equity, PRVs are considered a prime source of non-dilutive capital.

VC firm V-Bio said: “Reauthorization of the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) scheme has restored financial certainty and sparked intense interest from large pharma.”

Dan Williams PhD, chief executive of SynaptixBio, said: “The US dominates because the PRV program creates a highly valuable and, more importantly, tradable asset.

“VCs and private equity firms are far more willing to invest in rare disease biotechs simply because they provide a financial return on investment.”

Advertisement

He added: “While the UK is known for high-quality science and innovation, it has seen a sharp contraction in biotech fundraising. Without an equivalent to the FDA PRV program, UK rare disease biotechs rely heavily on public markets, private investment, or acquisition by larger global pharma to secure capital.”

The Association of the British Pharmaceutical Industry warned last September that the UK was slipping in the global race for life sciences investment, with foreign direct investment 58 per cent below 2017 levels.

There are signs of recovery at home. Figures from data platform Tracxn show UK life sciences funding rose 228 per cent to $3.2 billion in the first half of 2026, although the money went to fewer companies.

Market analysis published by Schroders in April said: “With public markets grappling with valuation volatility, the UK’s ‘golden triangle’ of innovation – spanning London, Oxford, and Cambridge – continues to produce the next generation of biotech champions.

Advertisement

“Historically, the UK’s Achilles’ heel has been the ‘Valley of Death’ – the gap between brilliant seed-stage science and the massive capital required for clinical trials. Too often, UK companies were forced to list in New York just to access the depth of capital needed to scale.”

Sergey Jakimov, founding partner at biotech VC firm LongeVC, told Cure: “Orphan therapies are already projected to be roughly a fifth of global prescription revenue. Pharma needs de-risked, clinically validated assets, and rare disease programs tend to show up better in diligence.”

The US has historically set the pace in rare disease drug development. The Orphan Drug Act of 1983 established incentives including market exclusivity, tax credits and support with getting into the clinic.

In the UK, the Medicines and Healthcare products Regulatory Agency published a draft rare disease therapies regulatory framework in May, designed to bring rare disease drugs to market more quickly. The consultation closed on 30 July.

Advertisement

Williams said: “It would be ideal if the UK could introduce a scheme similar to the PRV. With the proposed new framework we have everything in place to better manage the clinical trial and marketing authorisation process for rare disease therapies, but it stops there.

“Reducing regulatory and approvals timescales and costs can only be good for rare disease patients and their families, but adding this stronger incentive could transform the industry, making the UK a leading player in research and development in this key area.

“We still aim to conduct clinical trials in the UK, using the results to inform further trials in the US, but this all depends on raising further investment.”

SynaptixBio last year selected its lead candidate drug, an antisense oligonucleotide, for clinical trials. The technology silences mutated genes to stop them forming toxic proteins without altering the gene itself.

Advertisement

Around 1 in 17 people will be affected by a rare disease during their lifetime, more than 3.5 million people in the UK, but only around 5 per cent of the c10,000 known rare diseases have an approved treatment. Around 80 per cent are caused by a mutation in a single gene, making them more suitable for targeted treatments such as gene silencing.


Amy Ingham

Amy Ingham

Amy Ingham is a reporter at Business Matters, covering UK business news with a focus on breaking news, business policy, late payments and insolvency. She joined the magazine in 2026 after completing the NCTJ Diploma in Journalism at Harlow College’s journalism school. Her recent reporting includes British Steel’s nationalisation and its impact on SME suppliers, the decline in late payments by large firms, and Insolvency Service director disqualifications. Reach her at aingham@cbmeg.co.uk.

Advertisement
Continue Reading
Click to comment

You must be logged in to post a comment Login

Leave a Reply

Business

Despite supply concerns, world sugar prices keep steady

Published

on

Despite supply concerns, world sugar prices keep steady

But one analyst says there’s more upside than downside risk to the market.

Continue Reading

Business

Stock Market Today: Dow Rises On Iran Comments; SpaceX Rival Rocket Lab Dives

Published

on

Stock Market Today: Dow Rises On Iran Comments; SpaceX Rival Rocket Lab Dives

The Dow Jones Industrial Average and the other major stock indexes traded mixed Tuesday, after it was reported the U.S. and Iran had reached “some sort of an arrangement” for a peace deal. Meanwhile, SpaceX rival Rocket Lab (RKLB) was a big earnings loser on the stock market today. Just after Tuesday’s open, the Dow industrials rose 0.3%, as the…

Copyright ©2026 Investor’s Business Daily, LLC. All rights reserved. 87990cbe856818d5eddac44c7b1cdeb8

Continue Reading

Business

Solar eclipse chasers scramble for glasses as stocks run low

Published

on

A pair of eclipse glasses held up in front of the sun

Even just a small part of the Sun breaking through the glasses can harm your eyes, the Royal College of Ophthalmologists has warned, adding that the safest way to watch the event is indirectly – such as through a livestream or broadcast.

“Crucially, the retina has no pain receptors, so people will not feel the damage as it happens,” explains the college’s president Mohamed Elalfy.

The college reported around 70 cases of people experiencing vision problems after watching 1999’s eclipse – around 40% of those affected had looked at it for less than a minute.

Anyone who notices a change in their vision after looking at the Sun should seek advice from an eye-care professional.

Advertisement

If you cannot get your hands on proper glasses, the advice is to not simply opt for everyday sunglasses instead.

Imo Bell, a planetarium astronomer at The Royal Observatory Greenwich, says the specialist glasses are “very, very different” to regular sunglasses, which will not offer protection.

“They block out so much light that you can’t see anything through them except the Sun,” she told the PA news agency.

The UKHSA explains that regular sunglasses can also reduce glare and make it more comfortable for you to look at the Sun for longer – causing more damage.

Advertisement

You should not use binoculars, telescopes or the viewfinder of a camera even with your eclipse glasses on, because their magnifying effect can nullify the protection of the glasses and damage your sight.

For those without glasses or wanting to watch directly, you can do so using a pinhole projector.

You can make a simple one with a single sheet of card or something more ambitious using a cardboard box.

“We’re not going to get one this good for a really long time,” says Bell. “So, it’s important not to miss it, and to be prepared.”

Advertisement
Continue Reading

Business

Nearly 30,000 Pounds Of Argentine Beef Recalled In Florida And Texas Over Missed Import Inspection

Published

on

U.S. Department of Agriculture's Food Safety and Inspection Service, known

WASHINGTON — A Florida-based meat importer is recalling nearly 30,000 pounds of raw beef products brought in from Argentina after the shipment entered the United States without undergoing a federally required reinspection, agriculture officials said.

Corte Argentino USA LLC, based in Aventura, Florida, is recalling approximately 29,628 pounds of raw beef products that were imported from Argentina “without the benefit of import reinspection into the United States,” the U.S. Department of Agriculture’s Food Safety and Inspection Service, known as FSIS, announced Friday.

Under standard federal procedure, imported meat shipments must first clear U.S. Customs and Border Protection and the Animal and Plant Health Inspection Service before undergoing a separate reinspection by FSIS once they arrive in the country. That reinspection process examines shipping documentation, product labeling, packaging integrity and the general condition of the meat, and can include sampling for contaminants. In this case, the beef bypassed that final FSIS check before reaching distributors and retailers.

FSIS said the lapse was discovered during the agency’s routine inspection activities rather than through any report of illness or a foodborne outbreak investigation. The agency said there have been no confirmed reports of illness or injury linked to consumption of the recalled products.

Advertisement

The affected beef was produced between May 15 and May 20, 2026, and carries use-by or freeze-by dates ranging from September 15 to September 20, 2026. The recalled products were distributed to distributors and retailers across Florida and Texas, according to FSIS.

The recall covers several cuts of boneless beef packed in varying weights of cardboard boxes under the “FrigorIfico Gorina SAIC” label, including Top Sirloin Butt, marketed under its Spanish-language name “Cuadril Sin Tapa”; Eye Round, or “Peceto”; Topside Cap Off, or “Nalga AD S/Tapa”; Flat, or “Carnaza Cuadrada”; and Knuckle, or “Bola de Lomo.” All of the affected packaging bears the Argentinian establishment number “EST. N° OF. 2025” and the shipping mark “26644-AA,” details FSIS says consumers should check against any beef products they may have purchased from the listed lots.

FSIS said it is concerned that some of the recalled beef may still be sitting in consumers’ refrigerators or freezers, given the timing of the recall relative to the products’ extended shelf life. The agency is urging anyone who purchased the affected beef not to consume it, and instead to either discard the product or return it to the place of purchase for a refund.

Anyone who has concerns about illness or injury after consuming the recalled beef is being advised to contact a healthcare provider. FSIS also noted that it routinely conducts recall effectiveness checks following an announcement like Friday’s, verifying that the recalling company has properly notified its customers and confirming that the affected product has been removed from sale. A full retail distribution list for the recalled beef is expected to be posted on the FSIS website once it becomes available.

Advertisement

Consumers with general food safety questions can contact the USDA’s toll-free Meat and Poultry Hotline, while those looking to report a specific problem with a meat, poultry or egg product can do so through the agency’s online Electronic Consumer Complaint Monitoring System, which operates around the clock.

Friday’s recall adds to a steady stream of federal food recalls in recent weeks tied to a range of safety and regulatory issues. Just days earlier, more than 3,200 pounds of pastrami and corned beef products were recalled over possible listeria contamination, underscoring how frequently meat and poultry recalls occur even as reported illness rates connected to any single recall generally remain rare.

Import reinspection lapses like the one identified in Friday’s recall are considered a distinct category of food safety issue separate from contamination-driven recalls. Rather than indicating that a product is confirmed to be unsafe, a missed reinspection means federal verification of the shipment’s documentation, labeling and condition never took place as required, leaving the product’s safety status effectively unconfirmed by regulators at the time it entered the supply chain. FSIS has characterized such lapses as serious enough to warrant a full recall specifically because the agency cannot verify after the fact that the product met all import requirements, even in the absence of any specific evidence of contamination.

Corte Argentino USA LLC has not issued additional public comment beyond the information included in the FSIS recall notice. Consumers and members of the media with questions about the recall have been directed to contact the company’s general manager, Eial Kaplun, through information listed in the official FSIS announcement.

Advertisement

The recall is limited to the specific production dates, lot codes and shipping mark identified by FSIS, meaning not all Corte Argentino USA beef products are affected, only those matching the details tied to the flagged shipment. Consumers who are uncertain whether beef products in their possession match the recalled lots are advised to check packaging closely against the establishment number and shipping mark cited in the recall notice, or to contact their retailer directly for further verification.

FSIS has not indicated whether additional shipments beyond the identified lot are under review as part of the recall, and the agency said it will continue to provide updates on its website as the situation develops, including the retail distribution list once it becomes available for public review.

Continue Reading

Business

Diamond Hill Mid Cap Fund Q2 2026 Commentary (MUTF:DHPAX)

Published

on

Diamond Hill Mid Cap Fund Q2 2026 Commentary (MUTF:DHPAX)

Diamond Hill Capital Management, Inc. is a wholly owned subsidiary of Diamond Hill Investment Group, Inc. Diamond Hill Investment Group is a publicly traded company, and its shares trade on the NASDAQ (Ticker: DHIL). Note: This account is not managed or monitored by Diamond Hill Capital Management, and any messages sent via Seeking Alpha will not receive a response. For inquiries or communication, please use Diamond Hill Capital Management’s official channels.

Continue Reading

Business

10 Reasons Buyers May Want To Wait For Apple’s Rumored Foldable iPhone Instead Of Galaxy Z Fold 8 Now

Published

on

Apple's long-rumored foldable iPhone

Samsung’s Galaxy Z Fold 8 hit store shelves this month, but with Apple widely expected to unveil its first foldable iPhone within weeks, tech analysts say shoppers weighing a premium foldable purchase face a genuine timing question.

Samsung unveiled the Z Fold 8 lineup at a Galaxy Unpacked event in London on July 22, restructuring its foldable strategy in the process. The company split its flagship foldable into two models: a wider, more compact Z Fold 8 starting at $1,899.99 for 256GB, and a taller, more traditional Z Fold 8 Ultra starting around $2,099. Both became available for purchase in early August. Meanwhile, Apple has not yet confirmed its own foldable device, but a wide range of supply-chain analysts and industry reporters expect the company to unveil what is widely being called the iPhone Fold, or possibly iPhone Ultra, at a September event alongside the iPhone 18 lineup.

Here are 10 reasons analysts and reviewers say some buyers may want to hold off on Samsung’s device and wait to see what Apple brings to market.

First, Apple’s foldable is expected to arrive within roughly six to eight weeks of the Z Fold 8’s launch, according to multiple supply-chain reports, meaning buyers on the fence face a relatively short wait rather than a year-long gap.

Advertisement

Second, reporting from analysts including JPMorgan’s Samik Chatterjee points to a starting price of around $1,999 for Apple’s device, positioning it competitively against Samsung’s lineup rather than at a significant premium, based on currently available leaks.

Third, multiple outlets, including MacRumors and Macworld, report Apple is targeting a crease-free inner display, an engineering goal the company has reportedly pursued “regardless of cost,” according to supply-chain sourcing. Visible creases remain one of the most commonly cited complaints about existing foldable phones, including Samsung’s lineup, making a crease-free panel a potentially meaningful differentiator if Apple delivers on the claim.

Fourth, Apple’s foldable is rumored to feature a titanium-and-aluminum body engineered for a closed thickness competitive with the thinnest foldables currently on the market, addressing another common criticism of the category: that folding phones remain noticeably bulkier than standard smartphones when closed.

Fifth, buyers loyal to Apple’s ecosystem, including iMessage, FaceTime, AirDrop and continuity features across Mac and iPad devices, would gain access to a foldable form factor without leaving the platform, a factor that matters significantly for a large segment of premium smartphone buyers who have little interest in switching to Android regardless of hardware specifications.

Advertisement

Sixth, rumored specifications point to a roughly 7.8-inch inner display and 5.5-inch outer display for Apple’s device, dimensions comparable to Samsung’s more traditional Z Fold 8 Ultra model, meaning buyers would not necessarily sacrifice screen real estate by waiting.

Seventh, Apple’s foldable is expected to run on the company’s A20 or A20 Pro chip alongside a new Apple C2 modem, giving it access to Apple’s latest silicon rather than a chip shared with a prior generation of standard iPhones, according to spec leaks compiled by multiple outlets.

Eighth, some reports indicate Apple’s device will feature dual 48-megapixel rear cameras, a notable upgrade path for a first-generation foldable and one that would put its rear camera resolution on par with Samsung’s current flagship foldable offerings.

Ninth, Apple has historically waited to enter established hardware categories until it believes it can meaningfully differentiate its product, a pattern reflected in its approach to smartwatches, wireless earbuds and tablets. Analysts covering the foldable space have noted that Apple’s decision to enter the category years after Samsung suggests the company believes it has solved specific engineering problems, such as the crease and hinge durability, that have persisted across earlier generations of foldable phones from multiple manufacturers.

Advertisement

Tenth, buyers focused specifically on long-term software support may also factor in Apple’s typical multi-year update commitment for iPhones, which has generally extended longer than the support windows offered on most Android devices, including Samsung’s foldables, though Samsung has also expanded its own software support commitments in recent years.

Reviewers have also cautioned that waiting carries its own risks. Because Apple’s foldable would be a first-generation product, some analysts expect early units to face the kind of growing pains common to first-generation hardware, including limited initial stock tied to reported production yield issues and a smaller app ecosystem optimized for the folding form factor compared with Samsung’s more mature software experience, refined across eight generations of Z Fold devices since 2019.

Pricing also remains a genuine unknown for Apple’s device until the company confirms it directly. While a $1,999 starting price has been the most frequently cited figure across recent analyst notes, some earlier reports had suggested a range as high as $2,399, and Apple has not disclosed any details on trade-in or carrier promotions that could meaningfully offset the cost for buyers switching from an existing device.

For now, neither Apple’s final pricing, exact launch date, nor full specifications have been officially confirmed, and all comparisons between the rumored iPhone Fold and Samsung’s already-released Z Fold 8 remain based on leaks and analyst projections rather than confirmed details from Apple itself. Industry watchers say Apple’s September event, where the company is widely expected to unveil its full iPhone 18 lineup alongside its first foldable device, should resolve most of the remaining uncertainty for buyers deciding between the two ecosystems.

Advertisement
Continue Reading

Business

Earnings call transcript: Fennec beats Q2 2026 estimates as shares jump premarket

Published

on


Earnings call transcript: Fennec beats Q2 2026 estimates as shares jump premarket

Continue Reading

Business

FDA proposes to redefine GRAS

Published

on

FDA proposes to redefine GRAS

If enacted, the proposal would do away with self-affirmed process.

Continue Reading

Business

Microsoft Vs. AMD: Investors May Be Watching The Wrong Variables (Panel Regression) (MSFT)

Published

on

Microsoft Vs. AMD: Investors May Be Watching The Wrong Variables (Panel Regression) (MSFT)

This article was written by

I’m a seasoned financial analyst with a passion for puzzling out the complexities of the financial world. As a former writer for Fade The Market on Seeking Alpha, I diligently worked to provide insightful analysis and well-researched articles on various investment opportunities. However, I am no longer involved in analyzing, submitting, or commenting on articles for Fade The Market. With a vast experience, I have honed my expertise in evaluating market trends, analyzing investment opportunities, and providing strategic recommendations to optimize financial portfolios.

Analyst’s Disclosure: I/we have a beneficial long position in the shares of MSFT either through stock ownership, options, or other derivatives. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Seeking Alpha’s Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. Any views or opinions expressed above may not reflect those of Seeking Alpha as a whole. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank. Our analysts are third party authors that include both professional investors and individual investors who may not be licensed or certified by any institute or regulatory body.

Advertisement
Continue Reading

Business

Earnings call transcript: AECOM Q3 2026 revenue tops forecasts, EPS misses

Published

on


Earnings call transcript: AECOM Q3 2026 revenue tops forecasts, EPS misses

Continue Reading

Trending

Copyright © 2025