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There is a new most expensive drug ever—a gene therapy that costs as much as a Brooklyn brownstone or a Miami mansion, and more than the average person will earn in a lifetime.

Lenmeldy is a gene treatment for metachromatic leukodystrophy (MLD) and was approved in the U.S. on Monday. Its maker, Orchard Therapeutics, said today the $4.25 million wholesale cost reflects the value the treatment has for patients and families.

No doubt, MLD is awful. The nerve disorder strikes toddlers, quickly robbing them of their ability to speak and walk. Around half die, the others live on in a vegetative state causing crushing burdens for families.

But it’s also incredibly rare, affecting only around 40 kids a year in the U.S. The extreme rarity of such diseases is what’s behind the soaring price-tags of new gene therapies. Just consider the economics: Orchard employs 160 people, much more than the number of kids they’ll be able to treat over several years.

Giovanni in his Gene Therapy room on last day 1 jpg
A child in isolation after gene therapy for metachromatic leukodystrophyAMY PRICE

It means even at this price, selling the newest DNA treatment could be a shaky business. “Gene therapies have struggled commercially—and I wouldn’t expect Lenmeldy to buck that trend,” says Maxx Chatsko, founder of Solt DB, which gathers data about biotech products..  

Call it the curse of being the world’s most expensive drug.

The MLD therapy was approved in Europe starting three years ago, where its price is somewhat lower, but Chatsko notes that Orchard generated only $12.7 million from product sales during most of last year. It means you can count the number of kids who got it on your hands.

There’s no doubt the treatment is a lifesaver. The gene therapy adds a missing gene to the bone marrow cells of children, reversing the condition’s root cause in the brain. Many of the kids who got it, in trials that began in 2010, have been growing up to be beautifully average.

“My heart wants to talk about what an effect this therapy has had in these children,” says Orchard’s chief medical officer, Leslie Meltzer. “Without it, they will die very young or live for many years in a vegetative state.” But kids who get the gene therapy, mostly end up being able to walk and do well cognitively “The ones we treat are going to school, they’re playing sports, and are able to tell their stories,” Meltzer says.

Independent groups also think the drug could be cost-effective. One, called the Institute for Clinical and Economic review, and which assesses the value of drugs, said last September that the MLD gene therapy was worth it at a cost between $2.3 and $3.9 million, according to their models.

But there’s no denying that super-high prices can signal that a treatment isn’t economically sustainable.

One prior title holder for most expensive drug, the gene therapy Glybera, was purchased only once before being retired from the market. It didn’t work well enough to justify the $1 million price tag, enough to make it the price champion at the time.

Then there’s the treatment that’s been reigning as the costliest until today, when Lenmeldy took over. It’s a $3.5 million hemophilia treatment called Hemegenix, which is also a gene therapy. Such treatments were meant to be generate billions in sales, yet they aren’t getting nearly the uptake you’d expect according to news reports.

Orchard itself gave up on another DNA fix, Strimvelis, which was an out-and-out cure for a type of immune deficiency. It owned the gene therapy and even got it approved in Europe. The issue was both too few patients and the existence of an alternative treatment. Not even a money back guarantee could save Strimvelis, which Orchard discontinued in 2022.

Orchard was subsequently bought by Japanese drug company Kyowa Kirin, of which it’s now a subsidiary.

So it can seem like even though gene-therapies are hitting home runs in trials, they’re losing the ballgame. In the case of this Lenmeldy, the critical issue will be early testing for the disease. That’s because once children display symptoms, it can be too late. For now, many patients are being discovered only because an older sibling has already succumbed to the inherited condition.

In 2016, MIT Technology Review recounted the dramatic effects of the MLD gene therapy, but also the heartbreak for parents as one child

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By: Antonio Regalado
Title: There is a new most expensive drug in the world. Price tag: $4.25 million
Sourced From: www.technologyreview.com/2024/03/20/1089996/there-is-a-new-most-expensive-drug-in-the-world-price-tag-4-25-million/
Published Date: Wed, 20 Mar 2024 18:23:14 +0000

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How gamification took over the world

It’s a thought that occurs to every video-game player at some point: What if the weird, hyper-focused state I enter when playing in virtual worlds could somehow be applied to the real one?

Often pondered during especially challenging or tedious tasks in meatspace (writing essays, say, or doing your taxes), it’s an eminently reasonable question to ask. Life, after all, is hard. And while video games are too, there’s something almost magical about the way they can promote sustained bouts of superhuman concentration and resolve.

For some, this phenomenon leads to an interest in flow states and immersion. For others, it’s simply a reason to play more games. For a handful of consultants, startup gurus, and game designers in the late 2000s, it became the key to unlocking our true human potential.

In her 2010 TED Talk, “Gaming Can Make a Better World,” the game designer Jane McGonigal called this engaged state “blissful productivity.” “There’s a reason why the average World of Warcraft gamer plays for 22 hours a week,” she said. “It’s because we know when we’re playing a game that we’re actually happier working hard than we are relaxing or hanging out. We know that we are optimized as human beings to do hard and meaningful work. And gamers are willing to work hard all the time.”

McGonigal’s basic pitch was this: By making the real world more like a video game, we could harness the blissful productivity of millions of people and direct it at some of humanity’s thorniest problems—things like poverty, obesity, and climate change. The exact details of how to accomplish this were a bit vague (play more games?), but her objective was clear: “My goal for the next decade is to try to make it as easy to save the world in real life as it is to save the world in online games.”

While the word “gamification” never came up during her talk, by that time anyone following the big-ideas circuit (TED, South by Southwest, DICE, etc.) or using the new Foursquare app would have been familiar with the basic idea. Broadly defined as the application of game design elements and principles to non-game activities—think points, levels, missions, badges, leaderboards, reinforcement loops, and so on—gamification was already being hawked as a revolutionary new tool for transforming education, work, health and fitness, and countless other parts of life.

Instead of liberating us, gamification turned out to be just another tool for coercion, distraction, and control.

Adding “world-saving” to the list of potential benefits was perhaps inevitable, given the prevalence of that theme in video-game storylines. But it also spoke to gamification’s foundational premise: the idea that reality is somehow broken. According to McGonigal and other gamification boosters, the real world is insufficiently engaging and motivating, and too often it fails to make us happy. Gamification promises to remedy this design flawby engineering a new reality, one that transforms the dull, difficult, and depressing parts of life into something fun and inspiring. Studying for exams, doing household chores, flossing, exercising, learning a new language—there was no limit to the tasks that could be turned into games, making everything IRL better.

Today, we live in an undeniably gamified world. We stand up and move around to close colorful rings and earn achievement badges on our smartwatches; we meditate and sleep to recharge our body batteries; we plant virtual trees to be more productive; we chase “likes” and “karma” on social media sites and try to swipe our way toward social connection. And yet for all the crude gamelike elements that have been grafted onto our lives, the more hopeful and collaborative world that gamification promised more than a decade ago seems as far away as ever. Instead of liberating us from drudgery and maximizing our potential, gamification turned out to be just another tool for coercion, distraction, and control.

Con game

This was not an unforeseeable outcome. From the start, a small but vocal group of journalists and game designers warned against the fairy-tale thinking and facile view of video games that they saw in the concept of gamification. Adrian Hon, author of You’ve Been Played, a recent book that chronicles its dangers, was one of them.

“As someone who was building so-called ‘serious games’ at the time the concept was taking off, I knew that a lot of the claims being made around the possibility of games to transform people’s behaviors and change the world were completely overblown,” he says.

Hon isn’t some knee-jerk polemicist. A trained neuroscientist who switched to a career in game design and development, he’s the co-creator of Zombies, Run!—one of the most popular gamified fitness apps in the world. While he still believes games can benefit and enrich aspects of our nongaming lives, Hon says a one-size-fits-all

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By: Bryan Gardiner
Title: How gamification took over the world
Sourced From: www.technologyreview.com/2024/06/13/1093375/gamification-behaviorism-npcs-video-games/
Published Date: Thu, 13 Jun 2024 09:00:00 +0000

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Why we need to shoot carbon dioxide thousands of feet underground

This article is from The Spark, MIT Technology Review’s weekly climate newsletter. To receive it in your inbox every Wednesday, sign up here

There’s often one overlooked member in a duo. Peanut butter outshines jelly in a PB&J every time (at least in my eyes). For carbon capture and storage technology, the storage part tends to be the underappreciated portion.

Carbon capture and storage (CCS) tech has two main steps (as you might guess from the name). First, carbon dioxide is filtered out of emissions at facilities like fossil-fuel power plants. Then it gets locked away, or stored.

Wrangling pollution might seem like the important bit, and there’s often a lot of focus on what fraction of emissions a CCS system can filter out. But without storage, the whole project would be pretty useless. It’s really the combination of capture and long-term storage that helps to reduce climate impact.

Storage is getting more attention lately, though, and there’s something of a carbon storage boom coming, as my colleague James Temple covered in his latest story. He wrote about what a rush of federal subsidies will mean for the CCS business in the US, and how supporting new projects could help us hit climate goals or push them further out of reach, depending on how we do it. 

The story got me thinking about the oft-forgotten second bit of CCS. Here’s where we might store captured carbon pollution, and why it matters.

When it comes to storage, the main requirement is making sure the carbon dioxide can’t accidentally leak out and start warming up the atmosphere.

One surprising place that might fit the bill is oil fields. Instead of building wells to extract fossil fuels, companies are looking to build a new type of well where carbon dioxide that’s been pressurized until it reaches a supercritical state—in which liquid and gas phases don’t really exist—is pumped deep underground. With the right conditions (including porous rock deep down and a leak-preventing solid rock layer on top), the carbon dioxide will mostly stay put.

Shooting carbon dioxide into the earth isn’t actually a new idea, though in the past it’s largely been used by the oil and gas industry for a very different purpose: pulling more oil out of the ground. In a process called enhanced oil recovery, carbon dioxide is injected into wells, where it frees up oil that’s otherwise tricky to extract. In the process, most of the injected carbon dioxide stays underground. 

But there’s a growing interest in sending the gas down there as an end in itself, sparked in part in the US by new tax credits in the Inflation Reduction Act. Companies can rake in $85 per ton of carbon dioxide that’s captured and permanently stored in geological formations, depending on the source of the gas and how it’s locked away.

In his story, James took a look at one proposed project in California, where one of the state’s largest oil and gas producers has secured draft permits from federal regulators. The project would inject carbon dioxide about 6,000 feet below the surface of the earth, and the company’s filings say the project could store tens of millions of tons of carbon dioxide over the next couple of decades. 

It’s not just land-based projects that are sparking interest, though. State officials in Texas recently awarded a handful of leases for companies to potentially store carbon dioxide deep underwater in the Gulf of Mexico.

And some companies want to store carbon dioxide in products and materials that we use, like concrete. Concrete is made by mixing reactive cement with water and material like sand; if carbon dioxide is injected into a fresh concrete mix, some of it will get involved in the reactions, trapping it in place. I covered how two companies tested out this idea in a newsletter last year.

Products we use every day, from diamonds to sunglasses, can be made with captured carbon dioxide. If we assume that those products stick around for a long time and don’t decompose (how valid this assumption is depends a lot on the product), one might consider these a form of long-term storage, though these markets probably aren’t big enough to make a difference in the grand scheme of climate change. 

Ultimately, though of course we need to emit less, we’ll still need to lock carbon away if we’re going to meet our climate goals.  

Now read the rest of The Spark

Related reading

For all the details on what to expect in the coming carbon storage boom, including more on the potential benefits and hazards of CCS, read James’s full story here.

This facility in Iceland uses mineral storage deep underground to lock away carbon dioxide that’s been vacuumed out of the atmosphere. See all the photos in this story from 2022. 

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By: Casey Crownhart
Title: Why we need to shoot carbon dioxide thousands of feet underground
Sourced From: www.technologyreview.com/2024/06/13/1093635/carbon-dioxide-storage/
Published Date: Thu, 13 Jun 2024 10:00:00 +0000

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The Download: the rise of gamification, and carbon dioxide storage

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This is today’s edition of The Download our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

How gamification took over the world

It’s a thought that occurs to every video-game player at some point: What if the weird, hyper-focused state I enter when playing in virtual worlds could somehow be applied to the real one?

Often pondered during especially challenging or tedious tasks in meatspace (writing essays, say, or doing your taxes), it’s an eminently reasonable question to ask. Life, after all, is hard. And while video games are too, there’s something almost magical about the way they can promote sustained bouts of superhuman concentration and resolve.

For some, this phenomenon leads to an interest in flow states and immersion. For others, it’s simply a reason to play more games. For a handful of consultants, startup gurus, and game designers in the late 2000s, it became the key to unlocking our true human potential. But instead of liberating us, gamification turned out to be just another tool for coercion, distraction, and control. Read the full story.

—Bryan Gardiner

This piece is from the forthcoming print issue of MIT Technology Review, which explores the theme of Play. It’s set to go live on Wednesday June 26, so if you don’t already, subscribe now to get a copy when it lands.

Why we need to shoot carbon dioxide thousands of feet underground

Carbon capture and storage (CCS) tech has two main steps. First, carbon dioxide is filtered out of emissions at facilities like fossil-fuel power plants. Then it gets locked away, or stored.

Wrangling pollution might seem like the important bit, and there’s often a lot of focus on what fraction of emissions a CCS system can filter out. But without storage, the whole project would be pretty useless. It’s really the combination of capture and long-term storage that helps to reduce climate impact.

Storage is getting more attention lately, though, and there’s something of a carbon storage boom coming, as my colleague James Temple covered in his latest story. Read on to find out where we might store captured carbon pollution, and why it matters.

—Casey Crownhart

This story is from The Spark, our weekly climate and energy newsletter. Sign up to receive it in your inbox every Wednesday.

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 How Microsoft is building an AI empire
Its early investment in OpenAI helped it to leapfrog its old rival Google. (WSJ $)
OpenAI has lobbying regulators on its mind. (FT $)
Microsoft’s bet is paying off: OpenAI’s revenue has doubled. (The Information $)
Behind Microsoft CEO Satya Nadella’s push to get AI tools in developers’ hands. (MIT Technology Review)

2Rapid tests to target antimicrobial resistance are on the rise
Fast and easy analysis of common infections would stop doctors resorting to antibiotics. (FT $)
How bacteria-fighting viruses could go mainstream. (MIT Technology Review)

3 Stable Diffusion’s new release is generating horrifying bodies
Its mangled generations inspire revulsion and amusement in equal measure. (Ars Technica)
Text-to-image AI models can be tricked into generating disturbing images. (MIT Technology Review)

4 A hacker broke into Tile’s location tracking system
And they’re holding customer data to ransom. (404 Media)

5 Inside the lucrative black market for Silicon Valley’s stolen bicycles
🚲
One man made it his mission to unveil the theft pipeline. (Wired $)

6 What’s going on with Apple’s Vision Pro?
Analyst estimates suggest it hasn’t sold as well as expected. (NYT $)
It’s changing disabled users’ lives for the better. (NY Mag $)

7 Drone mapping is protecting slums from climate disasters
Because informal settlements aren’t visible on standard internet maps. (Bloomberg $)

8 The Excel World Championship is here
Spreadsheet fans, unite! (The Verge)

9 This humanoid robot can drive a car
🚗
That’s one solution to the problems posed by driverless cars. (TechCrunch)
Is robotics about to have its own ChatGPT moment? (MIT Technology Review)

10 America’s new cricket superstars are also tech workers
🏏
Saurabh Netravalkar, a software engineer for Oracle, is turning his hobby into a global spectacle. (WP $)

Quote of the

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By: Rhiannon Williams
Title: The Download: the rise of gamification, and carbon dioxide storage
Sourced From: www.technologyreview.com/2024/06/13/1093711/the-download-the-rise-of-gamification-and-carbon-dioxide-storage/
Published Date: Thu, 13 Jun 2024 12:10:00 +0000

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