CRISPR's Natural Blueprint Powers Gene‑Therapy Breakthroughs
How CRISPR works (in nature) and how it’s being leveraged as a tool for gene editing to cure and prevent diseases. Dr Alex Marson MD PhD of UCSF on the Huberman Lab podcast out now. https://t.co/fiL48JHxSL
GSK Secures Approval Yet Refuses to Produce Drug
$GSK is ostensibly the "sponsor" that won the approval but is once again saying it won't produce the drug.
BioNTech Founders Launch New Biotech, Eye FDA Acceptance
Sticking with the science. I find that really impressive. BioNTech founders Uğur Şahin and Özlem Türeci are leaving the biotech they founded to start a new one. Maybe by the time they have something for the clinic, the FDA will...
PCSK9 Drives Vascular Aging, Offers New Therapeutic Targets
PCSK9 in Vascular Aging and Age-Related Diseases Comprehensive summary of PCSK9's regulatory functions in vascular aging, highlighting potential therapeutic targets for combating age-related cardiovascular diseases. https://t.co/uo3PaSJLCb https://t.co/w8UZgIduPV
IPV Doesn’t Block Infection, but Stops Paralytic Polio
Actually he has it a bit backwards, those vaccinated with IPV can still acquire poliovirus and shed virus, but it prevents the virus from entering the bloodstream + protects almost 100% paralytic polio, just like the Covid vaccine protects 80-90%...
Vertex Drug Cuts Kidney Disease Marker in Late-Stage Trial
Vertex $VRTX says its drug successfully reduced marker of kidney disease in late-stage trial https://t.co/TbEkcHVYBB via @Jasonmmast
AI Predicts Asymmetric Cross‑coupling, Slashing Lab Time and Cost
A new AI-driven workflow predicts the outcomes of asymmetric cross-coupling reactions, enabling chemists to efficiently identify optimal molecular structures while significantly reducing laboratory time and costs. drugdiscovery
Bristol Myers Reports Positive Results for Next‑Gen Blood Cancer Drug
Bristol Myers claims success in study of another next-gen blood cancer drug https://t.co/fqPbZ2Szv8 by @gwendolynawu $BMY
FDA Drugs Show Promise as Future Geromedicines
Check out this article I was apart of @MensHealthMag where I discuss currently approved FDA drugs that are potential geromedicines and our work on centenarians. https://t.co/e95XvnY9fb
De‑risking Synthetic Biology: From Lab to Market
What does it actually take to bring a biological product to market? For more than 20 years, Ingenza Ltd has helped teams avoid the most common failure points in synthetic biology. Freedom to operate issues. Low productivity. Fragile processes. Late CMC...
FDA Draft Guidance Streamlines Low‑Cost Biosimilar Approval
New FDA draft guidance outlines important streamlining of path to getting low-cost biosimilars to the market - could reduce cost of pharmacokinetic studies and allow ex-U.S. comparitor products to be used for proving biosimilarity to U.S.-licensed drugs https://t.co/ErjNOga0qo
Digital Nudges Amplify Lifestyle Gains for GLP‑1 Patients
Medicines plus healthy habits create outcomes that neither could achieve alone. A new @StanfordMed study published in JAMA shows digital lifestyle nudges may catalyze a critical first step toward behavior change for patients on GLP-1 medications. “Achieving your best health involves a...
UniQure Sparks Gene Therapy Rally After Prasad Departs
UniQure leads genetic medicine biotech rally after news of Prasad’s exit https://t.co/zu1CtJfj29 by @realJacobBell $QURE $RGNX $ATRA $LXEO #GeneTherapy
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...
Multivitamin Modestly Slows Epigenetic Aging; Cocoa Doesn’t
Just published @NatureMedicine A daily multivitamin (MVM) slowed epigenetic aging in a randomized trial after 2 years; effect was small (~2 months) and not seen with cocoa extract supplement (vitamin was Centrum Silver) https://t.co/snOMNsTzW7 https://t.co/a6MhMuRhJb
Foundation Models Set to Transform Drug Discovery
Would you like to hear an opinion on how the future of drug discovery will look like as foundation models become more capable in drug discovery tasks? Check out our dialogue and the first pilot with the foundation model genius @ramin_m_h...
FDA Leaders Makary and Pazdur Meet Investors in Miami
Re: Prasad, from this morning's @statnews Readout newsletter: FDA Commissioner Marty Makary is in Miami this week speaking to health care investors attending a slew of broker conferences, noted Mizuho health care strategist Jared Holz. That should make for some...
Harnessing Immune System: New Frontiers in Cancer Therapy
The new Huberman Lab episode is out: Avoiding, Treating & Curing Cancer With the Immune System | Dr. Alex Marson 0:00 Alex Marson 2:21 Diseases & Current Biological Landscape; AI & Computational Tools 5:56 Immune System, Innate vs Adaptive Immune System 10:55 Thymus, T...
FMT Triples the Boost to Cancer Immunotherapy
Not just replication, but triplication of the benefit of revving up cancer immunotherapy with FMT (Yes💩) https://t.co/rXyQdO74FJ
FDA's Turbulent Leadership and Shift Away From Advisory Panels
Two terrific #FDA related pieces by @lizzylawrence.bsky.social & @matthewherper.bsky.social. Matt's is a look at lessons to be drawn from Vinay Prasad's chaotic tenure; Lizzy's delves into the FDA's turning away from adcomms. https://t.co/gC5GNTiPm1 & https://t.co/ygvylHi0uz
Five Key Lessons From Vinay Prasad’s FDA Tenure
. @matthewherper is so damn reasonable. Smart, too. 5 lessons from Vinay Prasad’s turbulent tenure at the FDA https://t.co/R65vPj5ep5
Undermanned VRBPAC Meets to Pick Flu Vaccine Strains
The #VRBPAC committee is scheduled to meet Thursday to advise #FDA on #flu vaccine strain selection. VRBPAC is supposed to have at least 15 members. It currently has 7 voting members & no chair. It has met only twice since...
TopTown SuperDNA Aims to End Aging by 2028
We will solve aging or will die trying. Check out the TopTown SuperDNA project. It is no longer a concept - projected launch in Q3 2028. Fully-integrated residential-biotechnology-clinical infrastructure is key to longitudinal AI-powered drug discovery. https://t.co/LK5XaTci29
RLMD's Strong Bladder Cancer Data Hits IBRX Valuation
The $RLMD bladder cancer data (phase 2) reported this morning look very good. Another competitive blow to $IBRX particularly at its bloated valuation. https://t.co/wYThfwIpG8
QURE-FDA Turbulence Highlights Market Volatility, MRNA Settlement Relief
9March: What a roller-coaster ride with $QURE and @US_FDA ! I sum up a wild week--plus $MRNA's could've-been-worse settlement with $ABUS & $ROIV (w/ insights from CEO Matt Gline) and updates from $DAWN and $TNGX in my latest StockWatch for...
Drug‑Safety Shock Hits Ipsen; Cut Exposure, Go Defensive
Macro: drug-safety shock; Key factor: SYMPHONY‑1 found secondary hematologic malignancies; Risk: regulatory re‑rating and pipeline impairment; Insight: trim Ipsen (IPN) exposure; favor defensive pharma 📉 — Viktor Kopylov, PhD, CFA. More insights: t.me/si14Kopylov
GSK Secures $300M Upfront for Linerixibat Deal
#GSK to get $300m upfront and $100m upon US FDA approval plus $20m for EU approval and up to $270m sales based payments from Alfasigma who will acquire rights to Linerixibat, a bile acid transporter inhibitor, currently under development.
Mitochondrial Quality Control Drives Senolytic Resistance
Comparative analysis of senolytic drugs reveals mitochondrial determinants of efficacy and resistance "findings suggest that mitochondrial quality control is a key determinant of resistance to ABT263-induced and ARV825-induced senolysis, providing a possible framework for rational combination senotherapies." https://t.co/xB0wFkzIW9
Vinay Prasad Mocked for Hosting Controversial HHS/FDA Call
Too funny. @emilyakopp (below) outs Vinay Prasad as the host of Thursday’s HHS/FDA media call to discuss $QURE.
OSK Gene Combo Reverses Joint Damage via Epigenetics
Osteoarthritis & back pain affects millions of people. Instead of managing symptoms, imagine rebuilding joints by making them young New study shows the reprogramming gene combo OSK regrows joints in mice & effect depends on TET2 so it's epigenetic...🧵 https://t.co/AlOIwcEXEQ
NR/NMN Lack Human Anti‑Aging Proof, Only PAD Benefits
stated as a critique but what does this mean as no drug has been demonstrated to have anti-aging effects in human clinical trials NR is active versus inflammaging and PAD, which is an age-related conditions affecting 10s of millions of americans you...
Consumer Biotech Set to Outgrow Disease‑Focused Pharma
GLP-1s already have market cap close to 1/3 of the top 10 biopharma companies combined. Consumer health biotech products will dwarf disease-focused biotech products.
Prasad’s Missteps as CBER Chief Spark Criticism
On Prasad’s last few weeks as CBER chief: 1. It was absurdly inappropriate to bring a large group of reporters together and rail on a pending application “on background” 2. The walk back of the Moderna RTF was a bad...
Patent Battles and GLP‑1 Turf Wars Prompt FDA Crackdown
Patent Fights, GLP-1 Turf Wars, and the FDA Tightens the Screws | Ep. 952 https://t.co/v0fulR3NfB https://t.co/9gGdkAVvlv
83% Unaware of DORAs: Sleep Knowledge Gap
There's a lot you can learn about sleep. Like 83% hadn't heard of DORAs. https://t.co/2lV3Fw6cwg https://t.co/IWRTdPTMOL
Cures for some Autoimmune Diseases Now a Reality
One of the most impressive advances we've ever seen for some autoimmune diseases: cures https://t.co/OZDuq4Iooy
AlphaFold Isn’t the Drug‑development Silver Bullet
Even with AlphaFold, protein structure is not a solved problem. And protein structure was never the bottleneck for drug development anyway. Let me explain.
Bitter Compounds Protect Brain in Diabetic Cognitive Decline
Bitter Compounds as Multifunctional Agents against Diabetes-associated Cognitive Dysfunction: Bridging Metabolic Regulation and Neuroprotection https://t.co/Xv3xItlP20
Seasoned Scientist Still Innovating Vaccines and Writing
Baylor did some new pics photos by my friend Agapito Sanchez Jr. Gosh I’m old, estoy viejo, but still making new vaccines, hope to have results of our hookworm anemia vaccine soon, hoping to reproduce our low cost Covid vaccine...
HGH May Harm Tendon‑Bone Healing Recovery
Human Growth Hormone May Be Detrimental When Used to Accelerate Recovery from Acute Tendon-Bone Interface Injuries https://t.co/L4EOFMKjys
Major Peptide Supplier Closes, Gray Market Faces Crackdown
Assuming this is real it may have something to do with their “must pay by Venmo to the following name” (at least that’s how it used to be). FDA is easing up on some aspects of peptides but gray market...
PEP Slows Aging by Blocking Inflammation Pathway
PEP protects against age-related inflammation, puts the brake on the aging process in mice, high levels in humans correlate with low inflammation So what is PEP, you ask? phosphoenolpyruvate, from glucose metabolism, a, master regulator, blocks cGAS-STING https://t.co/glMvaHSjOq https://t.co/MSVMBJbHm4
Roche, Zealand Stocks Tumble After Bland Obesity Drug Data
Roche, Zealand shares fall on ‘undifferentiated’ obesity drug results https://t.co/5pSR2vcHBD by @Lilah_Alvarado $RHHBY $ZEAL $LLY $NVO #obesity
Servier Spends $2.5B to Expand Cancer Pipeline
Servier to build cancer drug pipeline with $2.5B purchase of Day One https://t.co/aH0z8jt9tr by @realJacobBell $DAWN #biotech
Senior FDA Official’s Attack Sparks Debate Over Leadership
Gee, who could this "senior FDA official" be? It's a head scratcher. But he might tell Vinay Prasad that thinly veiled attacks against, oh, UniQure, and going off on advisory councils is a great way of keeping the political pot...
FDA's New Attack on UniQure Sparks Controversy
New from me on $QURE and FDA. Free, no paywall. The FDA, urged to avoid controversy, creates a new headache with attack against UniQure Anonymous diatribe from a senior official plunges agency back into headlines https://t.co/ceDE0TXjdI
B7‑H3 ADC HS‑20093 Shows Early Lung Cancer Activity
HS-20093, a B7-H3-targeted antibody-drug conjugate in lung cancer: Results from the ARTEMIS-001 phase 1a/b trial https://t.co/Jr90iovVeo https://t.co/Nl2WkprjU3
Exploring the Edge: 14 Innovators, 10 Biobanks
The edge is always weirder and more surprising than we can imagine, excited to follow the work of these 14 scientists and innovators. My personal favorite innovator is @ceeceeboom and her 10 biobanks 👀
Servier's $2.5B DAWN Deal Closes Ojemda Saga
$DAWN acquisition by Servier for $2.5bn is quite the ending for Ojemda. Who remembers this RAF inhibitor's bizarre & convoluted history? $BIIB $TAK https://t.co/uD5drO9ymr
Offshoring Drug Discovery Could Create Massive US Trade Deficit
Jake puts it crisply: >If discovery and early development move abroad, the nation risks locking in a massive trade imbalance: Foreign countries develop the drugs, and Americans pay the bills.