New gene-editing approaches for β-hemoglobinopathies
Nature Medicine, Published online: 17 April 2026; doi:10.1038/d41591-026-00021-7
Three phase 1/2 trials show that direct editing of HBG1 and HBG2 promoters is a promising disease‑agnostic strategy for treating β‑hemoglobinopathies such as sickle-cell disease and β-thalassemia.
A Digital Inclusion Intervention to Improve Access to a Digital Health Intervention Among Digitally Excluded Adults: Mixed Methods Pilot Randomized Controlled Trial
Trabectedin and low-dose irinotecan to target EWS::FLI1 in Ewing sarcoma: a phase 1/2 trial
Nature Medicine, Published online: 16 April 2026; doi:10.1038/s41591-026-04340-7
In this phase 1/2 trial, patients with Ewing sarcoma received trabectedin and low-dose irinotecan at concentrations known to inhibit the activity of the transcription factor EWS::FLI1, leading to encouraging clinical response rates.
STAT+: Roche to launch another Elevidys trial, with eyes on European approval
In an attempt to win European approval for the controversial medicine, Roche said Thursday it would run another trial of the Duchenne muscular dystrophy gene therapy Elevidys.
The Swiss company’s move comes after European regulators last year gave a negative review to the therapy, saying it had failed to demonstrate long-term benefits for patients with the degenerative muscle condition. Roche has rights to the therapy outside the U.S., where it is marketed by its developer, Sarepta Therapeutics.
Roche said the Phase 3 trial will generate the type of evidence that could lead to a resubmission with European officials and to applications with regulatory agencies in other parts of the world. The study will evaluate the safety and efficacy of Elevidys versus placebo over 72 weeks in roughly 100 boys at the early stages of the disease.
STAT+: Travere’s drug for a kidney disease doesn’t improve kidney function. The FDA approved it anyway
This is the online version of Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox.
Most of the recent conversations about the Food and Drug Administration have centered around the rejection of drugs for rare diseases that might have been approved had regulatory flexibility been applied.
This week, the FDA flexed its regulatory authority to approve the first medicine to treat focal segmental glomerulosclerosis, or FSGS, a rare disease in which scar tissue builds up in the filtering units of the kidneys, eventually leading to organ dysfunction and failure.
Effects of an Exercise-Assisting Mobile App (Osteoarthritis-Rehabilitation Assistant [O-RA]) on Rehabilitation Outcomes in Older Adults: Randomized Controlled Parallel Clinical Trial
Background: Mobile apps and biofeedback using motion analysis have both been used separately to increase compliance with exercise programs. We developed a mobile app, Osteoarthritis-Rehabilitation Assistant (O-RA), that uses motion analysis technology in the mobile app to assist older adults with performing a knee exercise program. Objective: This study aimed to evaluate the effects of the O-RA app on the compliance and correctness of the exercise program by older adults. Methods: We conducted an assessor-blind, parallel-design, randomized controlled trial with 40 older adults (aged 60 years or older) who had no symptoms and no diagnosis of knee osteoarthritis. Participants were divided into 2 groups: O-RA app (intervention) group and standard treatment (control) group. Both groups were taught 4 types of exercise programs by a physical therapist for 15 minutes and were instructed to do exercises at home every day for 1 week. The number of exercises, the percentage between observed and prescribed exercises, the correctness of exercises, and overall pain during the program were assessed in both groups. Results: The control group had significantly higher compliance with the exercise program than the intervention group (=3.5044, =.001). There was no statistically significant difference in the correctness of the exercise program between the intervention and control groups. The difficulty of use and satisfaction were 47 and 59, respectively, out of the full score of 100. The main problems were the instability and the difficulty using the app. Conclusions: In older adults without knee osteoarthritis symptoms or diagnosis, the O-RA app was not a facilitator but a barrier to the lower extremity exercise program. An updated version, aiming to increase the stability and make it more user-friendly, should be developed; however, more comprehensive data, including qualitative user feedback and standardized usability metrics, will be needed to effectively guide its design. Trial Registration: Thai Clinical Trial Record TCTR20240923002; https://www.thaiclinicaltrials.org/export/pdf/TCTR20240923002
<img src="https://jmir-production.s3.us-east-2.amazonaws.com/thumbs/c37817fc3e8476f49e094465f2f45581" />
Syncing Exercise to Body Clock Maximizes Cardiovascular Benefits
Timing exercise according to a person’s natural propensity towards being a “morning lark” or an “night owl” could maximize its cardiovascular benefits, a randomized trial suggests.
Matching exercise according to individual body clocks maximized the sleep quality and several parameters of cardiovascular health of middle-aged adults with preclinical risk factors.
The findings highlight the added value of incorporating circadian biology into exercise plans to optimize health outcomes.
Reporting their findings in Open Heart, the researchers suggest that assessing for chronotype—the predisposition towards morningness or eveningness—should be considered when prescribing exercise for those at risk of cardiometabolic disease.
“Our study shows that when you exercise may be just as important as how you exercise,” researcher Arsalan Tariq, PhD, from the University of Lahore, explained to Inside Precision Medicine.
“Aligning workouts with an individual’s biological clock significantly amplifies cardiovascular and metabolic benefits, offering a simple way to personalize prevention and improve adherence.”
A person’s chronotype affects their sleeping patterns, hormonal secretion, and energy levels during the day through an internal timing mechanism.
This is regulated by the circadian clock in a system that influences various physiological processes including blood pressure, heart rate, glucose metabolism, and vascular function.
Tariq and team examined how timing exercise affected key indicators of cardiovascular health among at-risk middle-aged adults.
Participants were aged 40 to 60 years and had at least one cardiovascular risk factor, such as high blood pressure, overweight, or obesity. The group also included those with a family history of premature cardiovascular disease.
Participants were randomly assigned to exercise at a time that either matched or did not match their chronotype, between 8am and 11am or between 6pm and 9pm.
This consisted of five, 40-minute sessions per week of supervised moderate intensity aerobic exercise such as brisk or treadmill walking for 12 weeks.
Of the 134 participants who completed the 60 sessions, 70 were larks—34 of whom had exercise matched with chronotype—and 64 were owls, with 30 matched to chronotype.
Measurements taken at the start of the trial and three days after it finished showed particular improvements in sleep and systolic blood pressure among those matched with chronotype.
Sleep quality improved by 3.4 points in matched participants versus 1.2 in the unmatched on the Pittsburgh Sleep quality Index. Systolic blood pressure dropped by 10.8 mmHg compared with 5.5mmHg in matched versus unmatched groups, respectively.
Chronotype-aligned exercise also led to significantly greater improvements in diastolic blood pressure, heart rate variability, peak oxygen consumption, low-density lipoprotein, and fasting glucose compared with misaligned exercise.
“Personalized, time-matched exercise interventions may become a practical strategy in clinical and public health settings, potentially leading to better outcomes and improved engagement,” the researchers reported.
“Future research and guidelines may consider circadian factors as a core component of lifestyle-based disease prevention.”
The post Syncing Exercise to Body Clock Maximizes Cardiovascular Benefits appeared first on Inside Precision Medicine.
STAT+: Novartis CEO joins Anthropic’s board
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Good morning. A reminder that if you’re ever feeling down about a mistake you made, there is always a way to turn it around — like how this delivery robot company has turned the issue of its robot crashing into my local bus stop into a marketing opportunity.
The need-to-know this morning
- Revolution Medicines raised $2 billion in concurrent stock and debt offerings. The mammoth financings — double what the company intended to raise — come just days after daraxonrasib, its experimental treatment for advanced pancreatic cancer, was shown to double the median overall survival of patients in a Phase 3 clinical trial.
Bain Capital again creates a startup with older pharma drugs
After Bain Capital last summer said it licensed five immunology drugs from Bristol Myers Squibb, it’s now unveiling the company to take those treatments forward: a startup called Beeline Medicines.
The Download: NASA’s nuclear spacecraft and unveiling our AI 10
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.
NASA is building the first nuclear reactor-powered interplanetary spacecraft. How will it work?
Just before Artemis II began its historic slingshot around the moon, NASA revealed an even grander space travel plan. By the end of 2028, the agency aims to fly a nuclear reactor-powered interplanetary spacecraft to Mars.
A successful mission would herald a new era in spaceflight—and might just give the US the edge in the race against China. But the project remains shrouded in mystery.
MIT Technology Review picked the brains of nuclear power and propulsion experts to find out how the nuclear-powered spacecraft might work. Here’s what we discovered.
—Robin George Andrews
This story is part of MIT Technology Review Explains, our series untangling the complex, messy world of technology to help you understand what’s coming next. You can read more from the series here.
Coming soon: our 10 Things That Matter in AI Right Now
Each year, we compile our 10 Breakthrough Technologies list, featuring our educated predictions for which technologies will change the world. Our 2026 list, however, was harder to wrangle than normal. Why? We had so many worthy AI candidates we couldn’t fit them all in!
That got us thinking: what if we made an entirely new list all about AI? Before we knew it, we had the beginnings of what we’re calling 10 Things That Matter in AI Right Now.
On April 21, we’ll unveil the list on stage at our signature AI conference, EmTech AI, and then publish it online later that day. If you want to be among the first to see it, join us at EmTech AI or become a subscriber to livestream the announcement.
Find out more about the list’s methodology and aims here.
—Niall Firth & Amy Nordrum
MIT Technology Review Narrated: this company is developing gene therapies for muscle growth, erectile dysfunction, and “radical longevity”
In January, a handful of volunteers were injected with two experimental gene therapies as part of an unusual clinical trial. Its long-term goal? To achieve radical human life extension.
The therapies are designed to support muscle growth. The company behind them, Unlimited Bio, also plans to trial similar therapies in the scalp (for baldness) and penis (for erectile dysfunction). But some experts are concerned about the plans.
Find out why the trial has divided opinion.
—Jessica Hamzelou
This is our latest story to be turned into an MIT Technology Review Narrated podcast, which we publish each week on Spotify and Apple Podcasts. Just navigate to MIT Technology Review Narrated on either platform, and follow us to get all our new content as it’s released.
The must-reads
I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.
1 Google, Microsoft, and Meta track users even when they opt out
According to an independent audit, they may be racking up billions in fines. (404 Media)
+ How our digital devices put our privacy at risk. (Ars Technica)
+ Privacy’s next frontier is AI “memories.” (MIT Technology Review)
2 OpenAI has a new cybersecurity model—and strategy
GPT-5.4-Cyber is designed specifically for defensive cybersecurity work. (Reuters $)
+ OpenAI has joined Anthropic in focusing on cybersecurity recently. (Wired $)
+ Like Anthopic, its latest model is only available to verified testers. (NYT $)
+ AI is already making online crimes easier. It could get much worse. (MIT Technology Review)
3 Amazon is buying satellite firm Globalstar in a bid to rival Starlink
The $11.6 billion deal targets the lucrative satellite internet market. (WSJ $)
+ Apple has chosen Amazon satellites for iPhone. (Ars Technica)
4 What it’s like to live with an experimental brain implant
Early BCI users explain what the technology gives—and takes. (IEEE)
+ A patient with Neuralink got a boost from generative AI. (MIT Technology Review)
5 Dozens of AI disease-prediction models were trained on dubious data
A few might already have been used on patients. (Nature)
6 Uber is breaking from its gig economy model to avoid robotaxi disruption
It’s spending $10 billion to buy thousands of autonomous vehicles. (FT $)
7 xAI is being sued over data center pollution
Musk’s AI venture stands accused by the NAACP of violating the Clean Air Act. (Engadget)
+ No one wants a data center in their backyard. (MIT Technology Review)
8 Apple could win the AI race without running
It may reap the rewards of everyone else’s spending. (Axios)
9 How 4chan set a precedent for AI’s reasoning abilities
The notorious forum tested a feature called “chain of thought.” (The Atlantic $)
10 The surprising emotional toll of wearing Meta’s AI sunglasses
Their shortcomings are making users sad. (NYT $)
Quote of the day
“Everything got a whole lot worse once they rolled out AI.”
—A copywriter tells the Guardian that they’re drowning in “workslop” — AI-generated work that seems polished but has major flaws
One More Thing
How refrigeration ruined fresh food
Bananas may not be chilled in the grocery store, but they’re the ultimate refrigerated fruit. It’s only thanks to a network of thermal control that they’ve become a global commodity. And that salad bag on the shelf? It’s not just a bag but a highly engineered respiratory apparatus.
According to Nicola Twilley—a contributor to the New Yorker and cohost of the podcast Gastropod—refrigeration has wrecked our food system. Thankfully, there are promising alternative preservation methods.
Read the full story on her research.
—Allison Arieff
We can still have nice things
A place for comfort, fun and distraction to brighten up your day. (Got any ideas? Drop me a line.)
+ Spotify only shows 10 popular songs per artist. This tool lists them all.
+ These GIF animations are mesmerizing loops of nostalgia.
+ This site beautifully visualizes Curiosity’s 13 years on Mars.
+ A retro-futurist designer has turned a NES console into a working synthesizer.

