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Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.

The treatment boosted levels of a protein in the study subjects' blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with.

The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.

"I'm very excited to see these results," says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR

But the results from the first 10 patients "represent an important scientific and medical milestone," says Dr. David Altshuler, Vertex's chief scientific officer.

All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they've had to undergo before getting the billions of edited cells infused into their bodies.

showed the gene-edited cells had persisted the full year — a promising indication that the approach has permanently altered her DNA and could last a lifetime.

"This gives us great confidence that this can be a one-time therapy that can be a cure for life," says Samarth Kulkarni, the CEO of CRISPR Therapeutics.

Gray has also been able to wean off the powerful pain medications she'd needed most of her life.

haven't needed the regular blood transfusions that had been required to keep them alive.

For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth. The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.

Doctors have already started trying to use CRISPR to treat cancer and to restore vision to people blinded by a genetic disease. They hope to try it for many other diseases as well, including heart disease and AIDS.

"This is really a life-changer for me," she says. "It's magnificent."

Beyond Crispr and Covid vaccines, there are countless potential applications of mRNA tools for other diseases; a new frontier for immunotherapy and next-generation cancer treatment; a whole new world of weight-loss drugs; new insights and drug-development pathways to chase with the help of machine learning; and vaccines heralded as game-changing for some of the world’s most intractable infectious diseases.

the vaccine innovations stretch beyond mRNA: A “world-changing” vaccine for malaria, which kills 600,000 globally each year, is being rolled out in Ghana and Nigeria, and early trials for next-generation dengue vaccines suggest they may reduce symptomatic infection by 80 percent or more.

the mRNA sequence of the first shot was designed in a weekend, and the finished vaccines arrived within months, an accelerated timeline that saved perhaps several million American lives and tens of millions worldwide — numbers that are probably larger than the cumulative global death toll of the disease.

As the first of their kind to be approved by the Food and Drug Administration, they brought with them a very long list of potential future mRNA applications: H.I.V., tuberculosis, Zika, respiratory syncytial virus (R.S.V.), cancers of various and brutal kinds.

A Nobel laureate, Doudna is known primarily for Crispr, the gene-editing Swiss Army knife that has been called “a word processor” for the human genome and that she herself describes as “a technology that literally enables the rewriting of the code of life.”

many of their back stories do rhyme, often stretching back several decades through the time of the Human Genome Project, which was completed in 2003, and the near-concurrent near-doubling of the National Institutes of Health’s budget, which helped unleash what Donna Shalala, President Bill Clinton’s secretary for health and human services, last year called “a golden age of biomedical research.”

A couple of decades later, it looks like a golden age for new treatments. New trials of breast-cancer drugs have led to survival rates hailed in The Times as “unheard-of,” and a new treatment for postoperative lung-cancer patients may cut mortality by more than half. Another new treatment, for rectal cancer, turned every single member of a small group of cases into cancer-free survivors.

Ozempic and Wegovy have already changed the landscape for obesity in America

although the very first person to receive Crispr gene therapy in the United States received it just four years ago, for sickle-cell disease, it has since been rolled out for testing on congenital blindness, heart disease, diabetes, cancer and H.I.V

all told, some 400 million people worldwide are afflicted by one or more diseases arising from single-gene mutations that would be theoretically simple for Crispr to fix.

in theory, inserting a kind of genetic prophylaxis against Alzheimer’s or dementia.

In January, a much-talked-about paper in Nature suggested that the rate of what the authors called disruptive scientific breakthroughs was steadily declining over time — that, partly as a result of dysfunctional academic pressures, researchers are more narrowly specialized than in the past and often tinkering around the margins of well-understood science.

when it comes to the arrival of new vaccines and treatments, the opposite story seems more true: whole branches of research, cultivated across decades, finally bearing real fruit

Does this mean we are riding an exponential curve upward toward radical life extension and the total elimination of cancer? No. The advances are more piecemeal and scattered than tha

“The biology and the science that we need is already in place,” he says. “The question now to me is: Can we actually do it?”

Sometimes these things just take a little time.

Classes will be online-only until further notice. Smart. But at some point parents will surely ask, “Why again are we paying 78 grand a year?” Is the end of universities far behind?

What about mobile and cloud computing, and even the stock market and its trillion-dollar valuations? It’s worth asking, as venture capital and private equity using cheap debt are keeping companies private longer, or forever

No, growth will still rule, but with a different set of leaders. In the bio world, DNA sequencing and Crispr gene editing are starting to ramp up.

Health care will be transformed by new ways to detect and treat cancer and other ways to cure previously incurable diseases like sickle-cell anemia.

Here’s hoping for some knock-your-socks-off new mobile products. Note also that we’re only about a third of the way into the cloudification of enterprises. And we’re only beginning to master machine learning and artificial intelligence, with their ability to find patterns that humans can’t. I think the next tech era will be driven by implementation of AI-infused systems into every business.

the past 30 year’s tech abundance means the developing world’s billions will finally see productivity improvements and attract an increasing share of investment. That’s probably right.

The shortfall in testing isn’t just a problem for individual patients and their doctors. It is also holding back large-scale surveys of seemingly healthy populations, in workplaces and elsewhere, and scientific research into fundamental properties of the virus and the disease it causes.

there is an escalating need to test much larger groups repeatedly—to track the spread of the virus as restrictions ease—and to carry out population-based studies that will reveal more about how this virus behaves.

in determining whether an individual is safe to enter a workplace or school on a given morning. Ideally, for the later purposes, tests would be conducted swiftly and at high volume at the places where samples are taken

All have served in one or more leadership roles: as presidents of universities or other academic institutions, as heads of government agencies, as advisers to drug or biotechnology companies, or simply as pioneers and mentors in their field. All have sought solutions to the great medical problems of our time. None of us can recall a crisis as stark as COVID-19.

While the need for greatly expanded testing in the next phase of this pandemic is widely acknowledged, the United States has no coordinated plan for how to achieve it. The technical building blocks are in hand, but how to put them together is not yet clear. Moreover, major regulatory hurdles limit the use of the results from novel tests in patient care, especially in certain states such as New York. And the logistics of deploying enough personnel to track samples and deliver results are daunting. Because of the complexity and importance of such testing, a centralized program, run by a strong scientific leader and paid for with federal dollars, may be the only solution.

rmed with efficient and accurate tests to detect the virus (indicating active infection) and reliable tests to measure antibodies against it (implying prior exposure and possible immunity), public-health programs could paint an accurate picture of the current pandemic. Small and large businesses, schools, health-care facilities, and other organizations could track the outcomes of their attempts to restore normal activities, and scientists could answer key questions about viral transmission and host immunity.

decisive answers will come only from studying human beings who are exposed to the virus under real-life conditions. Such studies may be feasible only under circumstances in which natural transmission is occurring at significant rates, as it currently is. Therefore, if we are to get answers to the following questions, we must act now.

tudies to answer these questions require identifying enough people who have recovered, then testing them repeatedly for the appearance of a new infection. Such people are relatively easy to find. They include doctors and nurses in hospitals in hard-hit metropolitan areas such as New York City; staff and residents at nursing homes with high rates of infection; and crews of U.S. Navy ships that have experienced outbreaks of COVID-19.

identify asymptomatic infections. Following up on those cases will shed light on how many asymptomatic people ultimately develop symptoms; how long it takes for them to do so; whether asymptomatic people who ultimately develop symptoms have higher viral loads than those who don’t get sick; whether symptomatic and asymptomatic people have different immune responses; whether other, simpler procedures (such as tests for some chemical abnormality in the blood) might be used to screen for infection; and how large a contribution asymptomatic people make to the ongoing transmission of the virus.

Despite repeated warnings after prior epidemics about the likelihood of new ones caused by novel microbes, the United States and many other countries failed to respond efficiently to this one. Scientists might have detected the new coronavirus much earlier with the better tools for microbial surveillance that already exist; prevented the pathogen’s worldwide spread by more aggressive testing and contact tracing; and supported better and safer health care with larger stockpiles and pipelines for procurement of medical equipment. Humanity should never be this unprepared again.

AI will usher in knowledgeable and friendly automated customer service any day now. But there is so much else on the innovation horizon: osmotic energy, geothermal, nuclear fusion, autonomous farming, photonic computing, human longevity. Plus all the stuff in research labs we haven’t heard of yet, let alone invented and brought to market.

Every industry is about to change, which will defy skeptics. Figure out how, and then, as Mr. Wozniak suggests, get your hands dirty. As always, the pain point is cost. Look for things that get cheaper—that’s the only way to clear the smoke and get new marvels into global consumer hands.

consider extreme poverty. It too has reached a record low, affecting a bit more than 8 percent of humans worldwide,

All these figures are rough, but it seems that about 100,000 people are now emerging from extreme poverty each day — so they are better able to access clean water, to feed and educate their children, to buy medicines.

If we want to tackle problems — from the war in Gaza to climate change — then it helps to know that progress is possible.

Two horrifying diseases are close to eradication: polio and Guinea worm disease. Only 12 cases of wild poliovirus have been reported worldwide in 2023 (there were also small numbers of vaccine-derived polio, a secondary problem), and 2024 may be the last year in which wild polio is transmitted

Meanwhile, only 11 cases of Guinea worm disease were reported in humans in the first nine months of 2023.

the United States government recently approved new CRISPR gene-editing techniques to treat sickle cell disease — and the hope is that similar approaches can transform the treatment of cancer and other ailments

Another landmark: New vaccines have been approved for R.S.V. and malaria

Blinding trachoma is also on its way out in several countries. A woman suffering from trachoma in Mali once told me that the worst part of the disease wasn’t the blindness but rather the excruciating pain, which she said was as bad as childbirth but lasted for years. So I’m thrilled that Mali and 16 other countries have eliminated trachoma.