Inside Moderna's Vision: Trust, Safety, and mRNA Future
Awesome to see @WSITYpod interview @moderna_tx president Stephen Hoge A great and wide reaching interview about trust, mRNA vaccines, spike protein, adverse events and what the future of mRNA technology is A candidate interview worth listening too.
AP-1 Activation Emerges as New Cancer Resistance Target
The knowledge of the many mechanisms by which cancer proves resistant to treatments keeps expanding. This week @Nature highlights activation of the AP-1 transcription factors, representing a new target https://t.co/UWSYDFM0IK https://t.co/6l4TISQonQ
Anemia May Heighten Alzheimer’s Risk Alongside P‑tau217
In evaluating p-tau217 and other biomarkers for risk of Alzheimer's disease, keep an eye on anemia, which may add to the risk, as seen in this new report https://t.co/ezbcLr4HIf https://t.co/TOKLRVBM4f
Human‑scale Data Aims to Close Drug Trial Gap
90% of drugs that enter clinical trials fail. A big part of why: the models they're based on weren't trained on human biology. Mouse data, non-representative cell lines, sparse perturbation coverage. The gap between in silico predictions and what actually happens...
Gut Microbiome Predicts Melanoma Recurrence Post-Immunotherapy
Gut microbiome markers predict recurrence of cancer after immunotherapy for metastatic melanoma @CellCellPress https://t.co/k5YuNnqfvt https://t.co/9UbuNAcYJh
Lilly's Obesity Pill Gains Momentum; OpenAI Launches Discovery Tool
Lilly’s obesity pill off to a strong start; OpenAI debuts new drug discovery tool https://t.co/upwygATKAv $LLY $GILD $JANX #biotech
Hands‑on PCA & CCA Tutorial for Cell Annotation
want to understand PCA projection and CCA for cell type annotation?my hands-on tutorial for cell type annotation is at https://t.co/lxePZCuypY https://t.co/hhrIQBcxll
YTE Poised as 2026's CRISPR-Like Biotech Breakout
YTE is to 2026 $XBI as CRSIPR was to 2021 $XBI $SYRE $ORKA $APGE https://t.co/CAuGPf5Hyk
Stable Weight Maintenance Predicts Longer Lifespan in Mice
Longitudinal analysis of body weight reveals homeostatic and adaptive traits linked to lifespan in diversity outbred mice "We observed that the ability to maintain stable body weight, despite fluctuations in energy intake and expenditure, was positively associated with lifespan in an...
Ribupatide Must Shine; Late GLP‑1 Faces Market Hurdles
All I'm going to say is that ribupatide needs to be a helluva drug for any of this to make sense. A me-too GLP-1 coming in a decade late seems like a big commercial challenge. $KLRA https://t.co/MAmA5f6FZ8
Metformin Misses Target in Type 1 Diabetes Trial
As a medical school professor, I love when medicine humbles us. Metformin has been around for a century. We thought we understood it. We were wrong. A new clinical trial gave metformin to people with type 1 diabetes -- not type 2....
Join Dr. Robert Califf on Future of Drug Regulation
We are so excited to host Dr. Robert Califf on April 20th, 2026, at 3pm EST. From leading the U.S. Food and Drug Administration to shaping the future of clinical research and drug regulation, Dr. Califf has been at the center...
Leiden University Seeks Postdoc for Next‑gen GPCR Drug Discovery
🚨 Hiring Postdoctoral candidate for next generation GPCR drug discovery Leiden University The Faculty of Science and the Leiden Academic Centre for Drug Research (LACDR) are looking for a:Postdoctoral candidate for next generation GPCR drug discoveryAre you excited about developing cutting-edge drug...
Kailera Raises $625M in Major Biotech IPO
From @gwendolynawu: Kailera nets $625M in one of biotech’s biggest-ever IPOs https://t.co/SJWx2QBmSn $KLRA #biotech $LLY $NVO $GPCR $VKTX #IPO
Review Panel Finds Anti‑amyloid Alzheimer Drugs Offer Little Benefit
Medical Review Group Questions Usefulness of Some Alzheimer’s Drugs Group says anti-amyloid treatments don’t offer noticeable benefits https://t.co/Z9tRQquyZX
GLP‑1 Drugs Show Promise for Treating All Addictions
GLP-1 medications get at the heart of addiction: study Diabetes and obesity drugs show promise in treating and preventing all substance use disorders https://t.co/buHywnw9Wk https://t.co/0lunbQbjsb
AI Creates Orexin Activator to Boost Focus, Reduce Sleep
Two researchers just used AI to design a selective orexin activator. If you care about focus, steady energy, or needing less sleep, this is worth paying attention to. https://t.co/3qbzB6J7X3
AI-Driven CRISPR‑GPT Enables Fully Autonomous Lab Experiments
A junior researcher walks up to a CRISPR experiment they've never run before. An AI agent has already decomposed the workflow, selected the guide RNAs, anticipated failure modes, and drafted the protocol. They run it successfully on their first attempt. That's not...
Mitochondrial Transplantation Reverses Cell Degeneration
In terms of my top bets for rejuvenation-based therapies, mitochondrial transplanation has entered the chat Cell-type-targeted mitochondrial transplantation rescues cell degeneration https://t.co/izvDaRk7kz
Cellular Stress Drives Stem Cell Aging, Revealing Therapy Targets
Beyond Cell Death: The Hidden Drivers of Stem Cell Aging “The findings shed light on how cellular stress shapes stem cell aging and highlight potential pathways for developing therapies to counter age-related decline...” https://t.co/hBUchsNtQ4 https://t.co/6HyOSZZ6ZS
Over 3,000 Brain Genes Show Sex‑Specific Expression
Sex-specific gene expression of the human brain, for >3,000 genes (of our ~20,000 total) @ScienceMagazine https://t.co/jQUFgARmuA https://t.co/0qNr9XPnP4
Choose the Right Design, Not One‑Size Truth
n=1 is can work for cancer and infectious disease, but often you need a clinical trial. We need to always be asking what the best tool is to get to truth. Don't assume that one experimental design is always correct. And don't...
P21⁺TREM2⁺ Macrophages Drive Inflammaging and Liver Disease
Delighted to be part of this study identifying p21⁺TREM2⁺ senescent macrophages as drivers of inflammaging and metabolic liver disease. A fantastic collaboration led by @ACovarrubiasPhD 👏
OpenAI Unveils GPT‑Rosalind for Pharma Drug Discovery
I truly believe OpenAI is hoping to drive a ton of revenue based on what it can do with health/medical -> OpenAI launches GPT-Rosalind, an AI model for life sciences research, including drug discovery, as a research preview for customers...
RFK Jr. Defends FDA Chief Amid Industry Backlash
RFK Jr. defends FDA, Makary following Republican questions - also defends the $REPL CRL: "Marty made the correct decision to not approve that drug. But everybody goes after him because the industry is so powerful." https://t.co/CuEeNFCYpp
Flawed Study Groups Failed and Successful Alzheimer Drugs Together
This new analysis of Alzheimer's drugs is such a good example of why we can't make any headway as a society. There is probably a good debate to be had on whether the risks and costs of the two approved beta...
High‑Resolution TORC2 Structure Opens Path to Age‑Related Therapies
Activating TORC2 holds potential in medicine for treating age-related memory & hearing loss. New study out today @MolecularCell reveals the structure of TORC2 in highest-ever resolution - which is good news for drug developers & all of us who age...
Drug Discovery Expands Beyond AI to New Frontiers
The marked expansion of paths and methods for new drug development including and beyond AI @Joseph_C_Wu @james_y_zou @WuXuekun @ScienceMagazine https://t.co/hTJ4Rt181n https://t.co/H3U5BNR1ER
1q22 Gain Predicts Poor Prognosis in Myeloma
Prognostic significance of acquired 1q22 gain in multiple myeloma [Oct 28, 2021] Hadiyah Y Audil et al. @myelomaMD Am J Hematol https://t.co/qUKCUdnZPH #mmsm #PrecisionMedicine #oncopath HT @EagleMyeloma https://t.co/vd0BVZ6bRq
1q Abnormalities Define Distinct Proteomic Landscape in Myeloma
Proteomic profiling revealed unique disease biology associated with 1q abnormalities in multiple myeloma [Apr 14, 2026] Mangalaparthi et al. @Nature_NPJ https://t.co/XTcJZIGFh6 #mmsm #PrecisionMedicine HT @Myeloma_Doc https://t.co/XyrxJ29elO
DNA Circles in Cancers Show Accelerated Epigenetic Aging
30 years ago, we discovered circular DNA molecules 1. pinch off yeast chromosomes 2. multiply 3. sequester epigenetic regulators (eg sirtuins) 5. cause aging New review today @CellCellPress about DNA circles in human cancers - which are epigenetically older than normal cells https://t.co/VlkBWCFnPi
From Static Snapshots to Dynamic Protein Modeling
AlphaFold solved protein structure prediction. That's a snapshot. But biology isn't static. Proteins flex, shift, and interact across time. Drug targets have multiple conformations. Cells don't act alone. The next hard problem is capturing that motion, and building models that reflect...
Scientists Propose Abandoning GLP‑1 as Obesity Target
Researchers behind GLP-1 obesity drugs advance new approach: Drop GLP-1 as a target https://t.co/ADzYb2Zqal via @statnews
ATAI Fully Acquires Ibogaine Program, Champions Psychedelic Shift
I am a strong believer in ibogaine, which is one of the reasons why @ataibeckley acquired the residual interest in its ibogaine program in Q4 2023 and now owns it 100%. I’m very encouraged to see the administration taking a positive...
FDA Pushes New
First autism, now low T - FDA continues to do its own literature searches and encourage sponsors to apply for new indications (never saw this in prior admins — with the exception of updating old cancer drug labels to include...
Replimune Downsizes While MeiraGTx Regains Eye Therapy
Replimune cuts staff; MeiraGTx reacquires eye gene therapy https://t.co/LqGPyNzRA3 $REPL $MGTX $JNJ $BIIB $RVMD $APLS
First Large Rapamycin Trial Aims to Boost Human Healthspan
Finally—this is the kind of study the field has been waiting for. A 720-person randomized clinical trial testing low-dose rapamycin in adults 65+ is about to get underway at @UAZPharmacy , with the goal of answering a simple but critical question:...
Third‑generation T‑cell Engagers Advance Cancer Immunotherapy
Cancer immunotherapy keeps revving up. Now onto a 3rd generation of T cell engagers https://t.co/h05f8h3i8L https://t.co/PP5XE8WWDr
Human Enhancement Is a Fundamental Right, Not Luxury
The United States Declaration of Independence enshrines the idea that every individual has the fundamental right to pursue a fulfilling and meaningful life. The government’s role is to protect that freedom - not to define happiness itself. I believe that one...
New Drug Extends Rheumatoid Arthritis Remission Years Post‑Treatment
Breakthrough Drug Delays Rheumatoid Arthritis for Years After Treatment Ends. https://t.co/03MjFzkPTM #research #RheumatoidArthritis #Arthritis #Pharma #healthcare
Eltrombopag Proves More Cost-Effective than IVIG Perioperatively
Cost-effectiveness of eltrombopag vs intravenous immunoglobulin for the perioperative management of immune thrombocytopenia [Jan 31, 2022] @ThisIsManraj et al. @BloodJournal https://t.co/2hr1CFmUro #ITP https://t.co/Fz6ZRjJHlk
CM313 Shows Promise as New ITP Therapy
A Novel Anti-CD38 Monoclonal Antibody [CM313] for Treating Immune Thrombocytopenia [Jun 19, 2024] Chen et al. @NEJM https://t.co/o9wf1ogUi4 #NCT05694767 #ITP https://t.co/aSXzpOHOtF
Fostamatinib Shows 79% Response, 54% Complete Remission in ITP
Fostamatinib [Syk inhib] effectiveness and safety for immune thrombocytopenia in clinical practice [August 8, 2024] @tomasjosegonza2 et al. @BloodPortfolio https://t.co/wrctRVBZsJ #ITP 79% RR, 53.6% CR https://t.co/OJfMY0DMah
Rilzabrutinib Shows Safety and Efficacy in Chronic ITP
Safety and efficacy of rilzabrutinib vs placebo in adults with [persistent/chronic] immune thrombocytopenia: the phase 3 LUNA3 study [Jun 12, 2025] Kuter et al. @BloodPortfolio https://t.co/hhnzXg3bqu #NCT04562766 #ITP https://t.co/IkjLnu1kTd
Daratumumab Shows Safety and Efficacy in ITP
Safety and Efficacy of Daratumumab in Immune Thrombocytopenia (#ITP) [Aug 5, 2025] Tsykunova et al. @BloodPortfolio https://t.co/GHyJGG7YSe #NCT04703621 https://t.co/pBgjzTCGQl
Ianalumab Combined with Eltromb
Ianalumab [VAY736] plus Eltrombopag in Immune Thrombocytopenia [Dec 9, 2025] @CukerMd et al. for the VAYHIT2 Investigators @NEJM https://t.co/99urXh8agT #NCT05653219 #ITP https://t.co/qKKhwv4ofR
Duchenne Kids Show Dramatic Improvement After Microdystrophin Treatment
A couple of weeks ago when I visited Solid Bio, they showed me some videos of Duchenne kids pre and post microdystrophin treatment that I was amazed by - but they weren't sharable yet. Bo just presented them at Needham...
Multi-Omics Integration: Complex Realities Beyond the Hype
Thread: Multi-omics sounds cool—until you actually try it. Here's are the nuances. 1/ You’ve got RNA-seq. Methylation. Proteomics. Time to “integrate” the data. But how? And why? Let’s break it down. https://t.co/Wxhcp6OJ68
Anti‑amyloid Drugs Show Trivial, Non‑meaningful Cognitive Benefit
The effect of current anti-amyloid drugs vs Alzheimer's disease is "trivial" for cognitive function or dementia severity, not clinically meaningful, from a systematic @cochranecollab review of 17 trials, >20,000 patients https://t.co/9A6PeZOyAt https://t.co/c1PCOYZxws
MGTX Buys Back XLRP Gene Therapy for $25 M
$MGTX just announced that it has entered into an asset purchase agreement with $JNJ to acquire back its bota-vec Gene Therapy program for the treatment of X-linked retinitis pigmentosa (XLRP) 🧵👇 for an upfront cash payment of $25M & a...