Decade-Long Quest to Break Blood-Brain Barrier Highlights Biotech Challenges
When we seeded Denali the idea was to break the curse of the blood/brain barrier. It took a decade and tons of faith and money. Biotech is hard. Curing diseases is hard. This is a good summary. https://t.co/qoJdWSLPsb
Asia Emerges as Testbed for Healthcare Innovation
It was a pleasure to participate in the Investment and Innovation Pathways to a Healthy Asia session at the Global Investors’ Symposium in Hong Kong. A timely discussion on where durable value is being created in healthcare across Asia, from prevention...
AI Empowers Chemists, Accelerating Novel Drug Discovery
The life of a chemist is about to change dramatically as we move away from tedious trial-and-error and deeper into the comfort of the prompt window. We don't need fewer medicinal chemists; we need more high-novelty drugs on the market...
Alpha‑Lipoic Acid Shows Promise for Ischemic Heart Failure
Efficacy of Alpha-Lipoic Acid in Patients With Ischemic Heart Failure: A Double-Blind, Randomized, Placebo-Controlled Study | @JACCJournals https://t.co/nW9SbwEDfy https://t.co/qsemjmMb9k
LLMs Enable DIY mRNA Vaccine for Pets
The coolest meeting I had this week with was Paul, who used ChatGPT and other LLMs to create an mRNA vaccine protocol to save his dog Rosie. It is amazing story. "The chat bots empowered me as an individual to act...
GLP‑1 Therapies Show Promising Cardiovascular Benefits
GLP-1 and the cardiovascular system "This Review summarizes the effects of GLP-1 and GLP-1RAs in the CV system..." https://t.co/Sy7Jjb96WD https://t.co/v3T01fIcOs
AI-Designed mRNA Vaccine Targets Cancer Precisely
Now imagine every cancer patient had access to this technology (they should) "A specifically designed mRNA vaccine - a precision-guided weapon to strike the cancer, developed with a pipeline designed by ChatGPT o1, implemented for candidate development by Gemini Pro 2,...
Billionaires Fund Headless Human Clones for Organ Farms
Billionaire-backed scientists aim to grow 'headless humans' to farm their organs... and help biohackers live for longer https://t.co/uQOzEtBapf https://t.co/HYZCMCOPy3
C1ORF112 Validated as Breast Cancer Driver After Early Prediction
Great to see a new work showing that C1ORF112 is upregulated in tumor tissues and that it promotes breast cancer proliferation. We computationally predicted C1ORF112 to be related to cancer back in 2012, and we were the first to study and...
Lancet Calls for Wider Access to GLP‑1 Drugs
This week's @TheLancet cover and editorial about getting GLP-1 drugs available for the people who need them the most https://t.co/BU9NZdmdGo https://t.co/TWOIKvfuth
MRD‑Negative Patients Can Stop Myeloma Maintenance Therapy
Discontinuation of maintenance therapy in multiple myeloma guided by multimodal measurable residual disease negativity (MRD2STOP) - @bdermanmd et al. @ajjakubowiak #ASCO24 Abstract 106 https://t.co/FBTY7SnCxK #NCT04108624 #mmsm #mmMRD
ODAC Backs MRD Testing as Early Myeloma Trial Endpoint
A Historic Turning Point: ODAC Unanimously Votes [4/12/24] in Favor of MRD Testing as an Early Endpoint in Myeloma Clinical Trials to Support Accelerated Approvals of New Treatments [Apr 18, 2024] @IMFmyeloma https://t.co/eDOgIrpVeR #mmMRD #mmsm #ctsm @FDAOncology https://t.co/ejA8KSoxOL
Clinicians Show Mixed Adoption of Myeloma MRD Testing
Clinician attitudes and practices toward measurable residual disease in multiple myeloma [Jun 7, 2020] @bdermanmd @jagoda_jasielec @ajjakubowiak Brit J Haematol https://t.co/8zhxYfYwZQ #mmsm #mmMRD https://t.co/lbZ4AFZ1YM
Insights on FDA, AI, and Misinformation with Rob Califf
What a joy to chat with my old friend Rob Califf @DrCaliff_FDA about the FDA under his leadership, the FDA today, AI regulation, misinformation, clinical research & more. We also discussed his memories of being a @UCSF medicine resident back...
Regeneration, Not Just Slowing, Could Reverse Aging
New Paper - Evidence suggests regeneration may be a natural and achievable biological process worth prioritizing over merely slowing aging—ideally beginning in midlife (around 40–60) to postpone decline, with the potential to reverse aging later in older individuals. Insights from...
Low‑cost, Patent‑free Vaccines Have Helped 100 Million
A “kazarian?” Is that like a Kardashian? What planet are these X trolls from? I develop low-cost often patent-free vaccines for global health and to help humanity. So far 100 million have benefited from access to our vaccine technologies developed...
AI Simulates Patient Tumors, Transforming Oncology Research
@Ronalfa is speaking at @SynBioBeta in May and you probably already know who he is if you've been paying any attention to the AI x bio space. Ron spent years at @RecursionPharma as SVP of Research and acting CSO, helping build...
New AI Platform Uncovers Hidden Cancer Targets, Raises $10M
Most cancer drugs go after the same targets: EGFR. PD-L1. HER2. Not because they’re the best targets. Because they’re the only ones we’ve been able to see. RyboDyn Inc. is going after what’s been invisible. The San Diego team, led by Imad Ajjawi, PhD,...
Cells Store Inflammation Memory via Epigenetic Chromatin Domains
🆕 @ScienceMagazine How do our cells have long term memory of inflammation that can later lead to persistent, chronic inflammation and disease? Through specific epigenetic chromatin changes, aka "memory domains" @ScienceVisuals https://t.co/mt70OwH144 https://t.co/DMk7SaIuBc https://t.co/FaPpN0331q
FDA Guidance Sparks Innovation Path for Inherited Disorders
My article in today's issue of the Journal of the American Medical Association Health Forum, on FDA's new rare disease guidance, and how the agency can build on these policy steps to promote innovation for inherited disorders, authored with Maarika...
FDA Approval Propels Denali, Boosts Rare Disease Biotech
FDA clears Denali drug in ‘clear step’ for rare disease biotechs https://t.co/x9yL4nyPaj by Kristin Jensen $DNLI #biotech
High‑Dose Psilocybin Outperforms Nicotine Patches Sixfold
One large psilocybin dose beat nicotine patches by 6x odds for smoking cessation. 82 otherwise-healthy cigarette smokers, 42 received a single high-dose 30mg/70kg psilocybin session, and 40 initiated an 8- to 10-week course of nicotine patch treatment. At 6 months; participants were 6x...
IPHYF Falls Short of Promise, Future Uncertain
Of all the IO companies I've met and covered in the past ~15 years I'm struggling to think of one that's been more disappointing vs its initial promise than $IPHYF. Will it even last beyond Q3? Via @ByMadeleineA -> https://t.co/DepdZfDBdY
Allogene's Interim Data Hints at Off‑Shelf CAR‑T Breakthrough
This week's Biotech Scorecard: Allogene $ALLO: A preview of its April interim analysis. Very interesting (to me, at least) There just might be a path forward for off-the-shelf CAR-T therapy in B-cell lymphoma. Frontline consolidation. Read all about it: https://t.co/tcPFelUH3C
Gene Therapy Turns Aging Into Editable Code
Gene therapy isn't just targeting diseases anymore—it's targeting aging itself. The hallmarks of aging are becoming editable code. Longevity Escape Velocity isn't a fantasy. It's an engineering problem. And we're solving it.
Harvard's Light Chip Enables Sorting Mirror-Image Molecules
Harvard’s twistable light chip could give drug developers a new tool for sorting mirror-image molecules https://t.co/WkjjD38exo https://t.co/5kr4d9f6sa
Eggs as Cheap, Scalable Factories for New Medicines
I have a story today about the quest to turn eggs into low-cost factories for medicines. Gift link: https://nyti.ms/4bzSabB
Code Ran Fine, but Biology Flagged Label Error
I almost sent a figure with wrong cluster labels to my collaborators. The R code ran perfectly. No errors, no warnings. I caught it because cluster 2 showed upregulated genes in RNAseq but reduced chromatin accessibility in ATACseq. Biologically, that's backwards....
BiotechTV Live Interviews at MassBio State of Possible
Pretty sweet setup for BiotechTv interviews at @MassBio State of Possible Conference today. If you are here, please stop by and say hello.👋 https://t.co/CQtXLi36Qk
Lifespan Extension Doesn't Guarantee Morbidity Compression, Study Shows
“These findings challenge the assumption that lifespan extension necessarily compresses morbidity, highlighting the need to consider lifespan, healthspan, and CoM as endpoints when evaluating anti-aging interventions. We do not claim that life-extending interventions categorically fail to achieve CoM; rather, we demonstrate...
Tech Accelerates Clinical Trial Recruitment of Ideal Participants
I spoke to a startup building software to help clinical trials recruit more of the right kinds of people more quickly - a serious challenge at present.
Anti‑TGFβ1 Antibody Shows Durable Efficacy, Low Toxicity
Scholar Rock's anti-TGFb1 antibody clinical study with early data in oncology indications... minimal AEs and signs of durable efficacy in checkpoint-refractory patients. $SRRK https://t.co/8xsN4wtbMV
Prescribing CORT Lifyorli Differs From Cushing’s Experience
Here's prescribing info for $CORT Lifyorli -> https://t.co/i46Whl5lX6 Why so different from the experience in Cushing's? https://t.co/EqvbCApqHn
Mini Brains Rewire to Balance Digital Pole
🔺 These Mini Brains Just Learned to Solve a Classic Engineering Problem 🧠 In a step toward biological computing, brain organoids rewired their networks as they learned to balance a digital pole on a cart. https://t.co/w2wHDTvIVp https://t.co/h51xTVlR4D
LEV Foundation Partners with Human Longevity Inc.
Delighted to share that LEV Foundation is initiating a collaboration with Human Longevity Inc. - details here: https://t.co/Ipfud85sGG
First Full GlyphAllo Data Reveal From Discovery to Human Proof‑of‑Concept
Proud of the work of our Seaport team & collaborators published today in @ScienceTM. This ~13 page peer reviewed paper is the first comprehensive data disclosure of our GlyphAllo™ program from discovery through initial human proof‑of‑concept.
Passion Fruit Compound Shields Mitochondria, Improves Mouse Memory
Alpha-amyrin, a molecule found in passion fruit, has demonstrated the ability to protect brain mitochondria and reduce memory loss in Alzheimer's mouse models, suggesting potential for future therapeutic development. neuroscience
Restarting SSRIs Improves Outcomes Over Med‑Free Approach
When we published our H2H in @NEJM Ian Jordan raised an excellent letter re SSRI discontinuation & how it might impact response. We looked & found he was right. Those who discontinued SSRIs and went back on (escitalopram) did far...
Microscopic Robot Delivers Targeted Drugs Inside Bloodstream
Microscopic #Robot Navigates the Bloodstream to Deliver Targeted Medication by @CeoImed #MedTech #Healthcare #HealthTech #Tech #Technology https://t.co/LBJeDdwTuq
Open Review Flags Flaws in Telomere‑river Longevity Study
The incredible study using "Rivers of telomeres" to rejuvenate tissues and extend lifespan in mice is now undergoing open peer-review. I did a review, pointing out issues with the methods and the lifespan curves. They're looking for more reviewers. https://t.co/KOiR4F85sT
Open-Source DIY Guide to Building mRNA Vaccines
Inspired by the man who built a personalized cancer vaccine for his dog, I’ve written an open-source guide to DIY mRNA vaccine production: philfung.github.io/openvaxx Drawing on my background in running lab startups, the guide covers the entire process - from sequencing...
Blueprint for Affordable, Accessible AAV Gene Therapy
When people ask "How can we make #AAV #GeneTherapy accessible and affordable" --> this is how 👇
New Review Links Β‑cell Stress to Type 1 Diabetes
Rethinking why the pancreas β-cells become vulnerable to autoimmune attack, leading to Type 1 diabetes. An illuminating review with ideas to counter β-cell stress @ScienceTM https://t.co/zn6a6wEg7u https://t.co/BV0LYnWRNL
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...
Escitalopram Yields -16.6 HAM‑D Drop After 8 Weeks
Anyone have a PDF of this? Perhaps there isn't a digital copy... This one showed an impressive -16.6 HAM-D drop with 8 weeks of escitalopram. It would be good to know their methodology. https://t.co/P5GCYcbxjk
Curiosity Fuels AGI: Autonomous Labs Powered by GPT‑5
Awesome being on the @Sequoia Training Data podcast with @gradypb and @sonyatweetybird. Topic links in the 🧵 The most human part of intelligence is curiosity, and science is formalized curiosity. If we want AGI — doing the work of science is...
Bridging Virtual Cells to Real Patients: The Next Frontier
Virtual biology burst onto the scene a few years ago. The idea was simple. Model the cell well enough, and you can predict biology before you test it. Large pharma has been thinking about this longer than most. PK/PD models, systems biology,...
FDA Clears Denali's AVLAYAH, 91% CSF
FDA clears Denali's AVLAYAH (macro), 91% CSF HS drop and analysts lift estimates (key). Risk: COMPASS confirmatory readout, infusion reactions. Trade: buy DNLI on 50‑day pullback 🚀 — Viktor Kopylov, PhD, CFA More insights: t.me/si14Kopylov
Morgan Stanley Sets $20 Target, 120% Upside for IMMX
$IMMX - Morgan Stanley initiates coverage with a $20 PT and an outperform rating, implying ~120% upside. https://t.co/Pd4uIH5BkI
Pharma AI Shifts From Cloud to On‑Device
Most AI companies believe the future is cloud-first. Pharma is different. In this industry, data is the kingdom. Experimental data. Molecular designs. Clinical insights. Some of the most valuable intellectual property on Earth. And in many cases, it cannot leave the lab. This is where the typical...
