Summit Sparks Breakthroughs: AI, Gene Therapy, CRISPR, mRNA
One thing that really makes the in-person summits we run at STAT so amazing: the people in the room. Here's what happened when we asked some of them for the last big breakthrough they saw. Featuring: the infectious @DrBlytheAdamson, genomcis pioneer @RobertGreene, former FDA CMO and ARCH VC @llborio, biotech leader @ksbosley, and the godfather himself, Stelios Papadopoulos. The answers: AI, gene and cell therapy combined with sequencing babies' DNA, a new take on mRNA vaccines, CRISPR, and data science in health. #STATBreakthrough
Stat News Sweeps SABEW Awards, Wins General Excellence
The SABEW "Best in Business" journalism awards just dropped. @statnews won for general excellence. @Jasonmmast won in the health/science category. @bobjherman won for best newsletters. Full list here: https://t.co/Nx9q71CLnP To my colleagues: https://t.co/aFDUhKyRFE
Merck Spends $6.7B on CML Drug Despite Gleevec Era
It's amazing that 25 years after Gleevec we are still watching drug companies pay billions for new drugs for CML. And that, in Adam's previous story, a once deadly cancer was referred to as a chronic disease. Anyway, $MRK buys $TERN...
FDA OKs Fecal Transplants, Yet Access Declines
Paradox: The FDA approved some fecal transplants for C. difficile. But accessing treatment got harder. https://t.co/AFFyCGPeKW
Lyme Vaccine Hits 70% Efficacy, Misses Confidence Threshold
The $PFE/ $VALN Lyme vaccine was a product for which there was a lot of hope. Results today show OK efficacy. Vaccine efficacy landed at 70%; investors hoped for 80% but thought as low as 60% would be relevant. But the...
Child's C. Diff Cure Required a Grueling Fecal Transplant Journey
Her son needed a fecal transplant for a fearsome C. diff infection. Getting one required a tortuous journey https://t.co/Ln54u1iTPV via @statnews
Pfizer Lyme Vaccine Effective yet Misses Crucial Statistical Benchmark
Pfizer’s Lyme vaccine shows efficacy, but misses key statistical hurdle There is a set up for drama here. $Valn $pfe Valneva stock is down 14% in premarket trading. https://t.co/WN0KOl4Nqh via @statnews
Blueprint for Building a $15B J&J Acquisition Target
How do you build a company that $JNJ will buy for $15 billion? Listen here.
Inside the CDC Director Search: Leadership Insights
If you care about the future of the CDC or public health, you need to read this piece and watch this video on how leadership is thinking about the search for a new director. Definitely watch the 10 minute video in...
Former FDA Chief Warns Political Pressure Plagued Trump Era
Political influence ‘a serious problem’ for FDA under Trump, former commissioner says https://t.co/52IR3LfECD via @statnews
Gene Therapy Delivers Real Results Amid Hype
Katrine Bosley: There's no question “hopes and aspirations” got ahead of the pace of any new science on gene editing #STATBreakthrough Seng Cheng: “The promise of gene therapy is correct. I think it has made that promise. and that's demonstrated by...
Early Experiments May Mislead; Models Need Validation
Jane Grogan from $BIIB again, on the fundamental challenge of drug discovery: do you believe what early experiments tell you? "We need to understand when [models] can be predictive and when they help at answering our hypothesis... “If you...
China Produces Triple U.S. PhDs, Boosting Biotech Innovation
$BIIB’s Jane Grogan on the impact of China on biotech innovation. “There's three times more PhDs that have been given last year in China than in the US. ... That's a lot of bright young things out there who are going...
Breakthroughs Take Years, Then Appear Overnight
$BMY chief scientist Robert Plenge: says there is “a joke” drug developers often repeat. “It's an overnight sensation a decade in the making. These things can actually be going on for a very long time, and then suddenly the field catches...
Profiting From Out‑of‑Network Bills, and the Fury It Sparks
How to get rich on out-of-network medical bills -- and how to get angry reading about it. By @TaraBannow https://t.co/fbmFyZhcic
Biology Leaders Tackle Alzheimer Research Reproducibility Crisis
For decades, they’ve set the record straight in biology. Next up: science’s reproducibility crisis A new coalition of biology grand challenge organizers will assess Alzheimer’s literature https://t.co/nLX1g0EEcB
Paralysis Patients Type Using Brain‑computer Interface
A brain-computer interface allowed people with paralysis to type with their minds https://t.co/W5icU6ye8c via @statnews
Judge Stalls Kennedy Childhood Vaccine Overhaul Meeting
Kennedy childhood vaccine overhaul stalled by judge The next meeting of the Advisory Committee on Immunization Practices was scheduled for this week, but has been postponed due to the ruling https://t.co/0cUIXEVgyV
Structure's Obesity Pill Shows Phase 2 Weight‑Loss Promise
Pretty impressive data from $gpcr, which remains my favorite ticker. Structure's obesity pill shows weight loss promise in Phase 2 trial https://t.co/T0JU2UuftY
MIT Professor Hired by RFK Jr Fails Scientific Standards
The MIT professor who has been appointed by Robert F Kennedy Jr to review the safety of Covid-19 vaccines has failed to meet basic scientific standards in his own research on the topic, according to more than a dozen scientists...
BioNTech Founders' Exit Needed as Sales Plunge
This morning's BioNTech news strikes me as very odd. Certainly, founder Ugur Sahin and Ozlem Tureci shouldn't feel compelled to run the company forever, and they may not be the right people to manage both the continued decline of Covid-19...
FDA Slashes Advisory Panels, Threatening Drug Safety Oversight
I've covered most of the big controversies the FDA has faced over the past 25 years: Vioxx, drug-coated stents, and Aduhelm all spring to mind. In all of these cases, the FDA's advisory panel system, in which the agency calls together...
FDA Veteran Decries Huntington Therapy Refusal as “Evil”
Amazing quote in this NYT story on the FDA from @By_CJewett. Janet Woodcock, a top drug regulator for decades, said reversals for a promising therapy send shock waves through the industry. “The Huntington’s refusal I thought was truly evil,” she told NYT....
Call for a Balanced, Transparent FDA Approach
My friend @adamfeuerstein delivers some great reporting and smart commentary on the FDA's direction that $QURE conduct a new randomized trial for its Huntington's drug. A senior FDA official and the company disagree on the basic question of whether the...
FDA Chief Backs Rare Disease Rejections, Lauds Vinay Prasad
FDA Commissioner Marty Makary defends rare disease drug rejections, Vinay Prasad "Vinay Prasad is a genius,” Makary said. “He’s on loan from UCSF. He’s published over 500 scientific peer-reviewed articles, and some of the drugs they are criticizing him for...
Galleri Test Misses Primary Goal, Shows Secondary Promise
A key study of $GRAL's Galleri test in the UK failed to reach its primary endpoint, but showed benefits on a secondary endpoint. Tell me what you all think. https://t.co/CDrvokqPop
Former Psilocybin Trial Participant Shocked by New Findings
"I took part in a 2012 psilocybin trial. What I’m seeing now horrifies me" https://t.co/T1BLv6n59X via @statnews
FDA Chief: Most Drugs Belong Over‑the‑counter
FDA chief Marty Makary says 'everything should be over the counter' unless drug is unsafe or addictive https://t.co/HiYBuxvyWC
Federal Vaccine Panel Meeting Delayed After HHS Missed Deadlines
Upcoming meeting of federal vaccine panel may be postponed after HHS missed deadlines https://t.co/zfAl7owoQg via @statnews
FDA Splits Moderna Flu Vaccine Review by Age
FDA agrees to review Moderna flu vaccine application, splitting review into standard approval for those under 65 and accelerated approval for those 65 and above. $mrna https://t.co/HMMlPm0PGh
FDA Rejects Review of Moderna’s Experimental Flu Vaccine Data
Here the Moderna flu data the FDA refused to review. This is the most important slide from the company's study of its experimental flu vaccine-- exactly the data FDA said it wouldn't even review. And below is a thread of...
Regeneron Unveils Next‑Gen Dupixent Amid Rising Competition
There's a fascinating fight brewing between Regeneron and, well, everybody else. But what do we call it? Dupixent, the treatment for atopic dermatitis and other autoimmine issues that $REGN sells with Sanofi, is one of the biggest selling drugs in the...
Moderna Swaps Vaccine Chief as Development Hurdles Rise
Another possible data point on the challenges for new vaccine development: Moderna's chief medical officer, Jacqueline Miller, a longtime vaccine developer who rose through the ranks at GSK and Merck, is departing. David Berman, a veteran of immuno-oncology efforts at AstraZeneca...
FDA Rejects Anktiva Expansion After Mischaracterized Meeting
My colleague @adamfeuerstein is reporting today that @DrPatrick "mischaracterized the outcome of a face-to-face meeting he had with FDA officials" to broaden the use of Anktiva, the bladder cancer drug that is sold by ImmunityBio, where Soon-Shiong is chairman. Adam reports...
Ex‑Vaccine Court Lawyer Now Guides
This Teen Never Got His Day in Vaccine Court. His Former Lawyer Now Advises RFK on Its Overhaul. This story complicates the simple. https://t.co/RSrad8WOnh
Hospital Group Challenges Eli Lilly's 340B Data Policy
Hospital group seeks to have new Eli Lilly policy on 340B claims data blocked https://t.co/Uz0lDrgL1y
Shared Vaccine Decision‑making Isn’t the Enemy, Says Sandman
I think you should always listen to Peter Sandman, even if you don't agree. "Shared decision-making on vaccines is not the enemy," one of the great experts on medical decision-making writes in STAT. https://t.co/1XZnI9JZ1m
Remember William Foege: Smallpox Eradication’s Rational Hero
I don't know how to ask for a moment of silence amid the madness that is X. But please take a moment to reflect on the heroism of William Foege. Foege, who died Saturday at 89, was a key architect of...
ACIP Chair Calls for Reassessing Polio Vaccine Necessity
So we are the point of questioning polio vaccines. The chair of ACIP: “I think also, as you look at polio, we need to not be afraid to consider that we are in a different time now than we were then....
Bipartisan Deal Paves Medicare Path for Grail's Cancer Test
In case you missed it, one apparent winner in today's bipartisan healthcare deal is the diagnostics firm Grail. $GRAL The company’s "multi-cancer early detection" test is already available, but in order to succeed it must leap multiple hurdles, including receiving...
RFK Jr. Seeks Overhaul of Vaccine Compensation Panel
RFK Jr. is moving to remake a panel involved in that compensating people who are injured by vaccines. It could have big consequences. By @ChelseaCirruzzo. https://t.co/S98hMN6Vd0
FDA's Regulatory Firewall Cracked, Staff Faces Trauma
Conducting interviews on stage can be emotional. Last night I got to speak with Richard Pazdur, the long-time FDA cancer czar who recently retired, along @ScottGottlieb and former FDA official Julie Tierney. Pazdur changed the way medicines are regulated, getting them...
U.S. Becomes Outlier in Global Vaccine Schedules
Fact check: When it comes to vaccine schedules, the U.S. is now the outlier STAT analysis shows top officials have put the country out of step with peer nations By @HelenBranswell https://t.co/t8ZmqatU1U
Baby KJ Scientist Launches Aurora Therapeutics for Personalized CRISPR
Key scientist from Baby KJ team launches startup to scale personalized CRISPR medicines Aurora Therapeutics is one of several efforts vying for space in the growing field Great writing from @Jasonmmast and @ADeAngelis_bio https://t.co/kbDJxY6Sq5
Parabilis Secures $305M, CEO Signals IPO Plans
Mathai on quarterly conference calls? Parabilis Medicines raises $305 million as CEO warms to an IPO By @ADeAngelis_bio https://t.co/h6I9BXDuLS via @statnews
