Makary Misrepresents Unresolved Drug Applications, Conflates Therapies
(Hopefully) Final word today on the Makary $QURE (or was it $RGNX) thing: Bottom line: Makary shouldn't be talking publicly about unresolved drug applications. Uniqure doesn't even have an application submitted; and RegenxBio's CRL letter isn't posted to the FDA's web site. Also, does Makary even know what the hell he's talking about? "...there was a product where the researchers drilled a burr hole, literally a hole in people's skulls..." That's Uniqure, clearly. But... "...inject intrathecally into the ventricle..." could be RegenxBio, which is injected into the spine. Is Makary conflating two different therapies? Total shitshow...
Biotech VC Webinar Launches with Top Experts, Thousands Registered
We'll be gearing up today's webinar on biotech venture capital at the top of the hour. Great guests, great topics. If this is a central issue in your life, tune in. Thousands have signed up already. https://t.co/c16y04mFsD
Makary Slams FDA, Defends Rare Disease Drugs Amid Controversy
You've got to watch this Makary interview on CNBC. He attacks a drug, hits the Old FDA and circles all the wagons around Vinay Prasad, who "loves President Trump" and faces a fatwa (WSJ). Makary is getting major props for...
DIY Gut Microbiome Tests Are Unreliable and Flawed
Direct-to-consumer gut microbiome assessments are unreliable, problematic: a review of 7 companies with significant methodologic issues https://t.co/atwmZUNjVE https://t.co/SqlMljuW0y
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...
FOCAS Enables Precise Site‑Specific M6A Erasure via CRISPR
m6A is the most common internal mRNA modification, but we still don't know what most m6A sites actually DO. Zhang et al. built FOCAS -- a CRISPR-based platform using dCas13b-FTO to precisely remove m6A at specific sites without touching the DNA. https://t.co/G6kSgpSSbV...
Startups Must Verify CRISPR Patent Freedom Before Launch
Anyone who uses CRISPR systems outside of non-profit and research purposes is subject to the patents that claim those processes as theirs. A lot of startups forget to do due diligence on that and think its fair game. Have you...
Learn 6 Plot Types to Replicate Any Genomics Figure
Master these 6 types of plots to reproduce any genomics paper figures. I will prove it at the end of the video. https://t.co/gjRs0gY6Mx
Orforglipron Outperforms Other Oral GLP‑1s in T2D Trial
Head-to-head trial of the oral GLP-1 drugs in people with T2D. Orforglipron was superior https://t.co/9B7RXIgdJY
FDA Chief Warns Against Approving Risky, Ineffective Rare‑disease Drugs
$QURE down this morning on comments made by FDA Commissioner Marty Makary to CNBC's Becky Quick. In a discussion about rare-disease drug approvals and Vinay Prasad, Makary said this: "I think there has been a bit of an effort to...
Progress Toward Off‑the‑Shelf Vaccines for Hereditary Cancer
We're making headway for off the shelf preventive cancer vaccines in people with hereditary forms of cancer, e.g. Lynch syndrome https://t.co/F3LqKS46BQ
OrbiMed Leads Top Five Biotech VC Dealmakers
Always fun to look over @JohnCendpts annual list of the top 100 most active VCs in biotech for the past year Top 5, by # of rounds: 1. OrbiMed 2. RA Capital 3. GV 4. General Catalyst 5. Catalio Full list & analysis is live...
NTLA Floods Market with 6M New Shares, Cash‑burn Evident
$NTLA looks like they ran the ATM for nearly 6M new shares since last quarterly earnings? As I expected. Absolute cash furnace. Their runway guidance was always mathematically implausible without raising like this.
RVMD Switches PRMT5 Partner From T
$RVMD was collaborating with $TNGX in PRMT5, but now it's working with $BMY (remember that ex MRTX project?). Via @ByMadeleineA @ApexOnco -> https://t.co/mGZjKOiB7p (And $AMGN still dose optimising.)
GLP‑1 Drugs Plus Healthy Habits Cut Heart Events
The combination of GLP-1 drugs (such as Ozempic) and healthy lifestyle factors was associated with less major adverse cardiovascular events in ~100,000 people with T2D @TheLancetEndo https://t.co/phNzEBy7zY https://t.co/DDVi8uedRP
Psychedelic Stocks Overhyped as Nominee Warns Caution
Psychedelic tape still trades on vibes > policy. Surgeon General nominee Casey Means says she “would not recommend” psilocybin to the American people today. Yes, she nods to “exciting work” in PTSD and veterans. But that is a far cry from...
FDA Rejects Experimental Rare Blood Cancer Therapy
An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year — with internal reviewers recommending it be cleared — before the agency rejected the drug last month, @adamfeuerstein...
FDA Rejects Europe‑approved Drug: What Shifted?
Interesting read: the thread and the story. A drug that was approved in Europe in 2022, which looked like it was on track for #FDA approval, instead is rejected. What changed at FDA? 🤔
ACIP Meeting Notice Raises Questions on Vaccine Injury Role
The Federal Register notice for the (now) March #ACIP meeting is now up & it's ... puzzling. Possible vote on Covid vaccine injury? ACIP can recommend changes to the way a vaccine is used, for safety reasons. But vaccine injuries aren't...
AI Should Boost Human Response Rates, Not Invent New Drugs
$LTRN is a live experiment in our thesis: AI doesn’t need to invent new drugs. It needs to improve probability of success in humans. If RADR consistently enriches responders, that’s durable value. If not, platform premium disappears. Listen to the podcast here: https://t.co/ZAg5btpcIK
Dual BCMA‑CD19 Targeting Shows Promise in ASyS
interesting and different efficacy outcomes in ASyS and SSc autoimmune patients treated with blinatumomab (CD3/CD19) and teclistamab (CD3/BCMA), respectively, + Rituximab maintenance. Potential for targeting both BCMA and CD19, especially in ASys. https://t.co/Dyq7h9EeGo
AI‑Engineered AAV Capsid Makes Gene Delivery Programmable
One company is unlocking gene delivery to overcome the challenges that held back the promise of genetic medicine. If you want to learn more, they’ll be hosting an exclusive fireside chat at 9am Thursday, May 7th at SynBioBeta. Last year at...
Stem Cell Therapy Boosts Frailty Walk Test Performance
A randomized trial of a stem cell therapy vs placebo for aging frailty showed durable and dose-dependent improvement in the 6-minute walk test @CellStemCell https://t.co/2jOYuIgUrY https://t.co/04mEV7OdLo
Identifying Key Barriers to Boost Myeloma Trial Enrollment
Identification of Significant Barriers to Accrual (BtA) to @theNCI Sponsored Multiple Myeloma – Clinical Trials (MM-CT): A Step towards Improving Accrual to #ClinicalTrials - @mweissmdphd et al. #ASH12 Abstract 3165 https://t.co/i4zO7ktqHJ #mmsm #caxtx #ctsm
Quadruplet Therapy Effective without Early Transplant in NDMM
#EAOnc EQUATE EAA181 Effective Quadruplet Utilization After Tx Evaluation: Ph3 RCT NDMM Not Intended for Early ASCT [Activated: 10/27/20] PI= @myelomaMD https://t.co/qD26HMx3J8 #NCT04566328 #mmMRD #mmsm @eaonc @mweissmdphd @VincentRK @LynneWagnerPhD Wei Snyder Kostakoglu @mtmdphd
Phase 3 Trial Tests Daratumumab Regimens for New AL Amylo
.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/uikUnnJd3n
Super Agers Generate More Neurons Than Much Younger Adults
New @Nature The brains of Super Agers produce more neurons than people some 40 years younger, a neurogenesis resilient signature linked to exceptional cognitive health and memory skills https://t.co/tNB0dTea2F
PI‑Rd Triplets Show Superior Efficacy And
Comparative Effectiveness and Safety of PI-Rd Triplets in Relapsed/Refractory Multiple Myeloma: INSIGHT-MM Data Analysis [Jan 13, 2026] @NoemiPuigM et al. European Journal of Haematology https://t.co/Vf7USecLlR #NCT02761187 #mmsm #openaccess https://t.co/C5Ya6TTQfo
Combining EMR and Multi‑omics Sharpens Biological Age Predictions
Assessment of biological age integrating electronic medical records and multi-omics increases accuracy for predicting outcomes @NatureAging https://t.co/7y60SSPTQf https://t.co/u02OlWMdQz
Emerging Protein Degradation Trends and CAR‑T Synergies
What gets missed in the hurly burly of a conference with many highlight reels? Here we explore emerging trends to watch out for when it comes to protein degradation/glues, & combination opportunities w/ CAR-T cells. Time for some...
Bioinformatics Needs Git: 6 Essential Commands
1/ If you're doing bioinformatics without Git, you're gambling with your research. Here are 6 Git commands every bioinformatician must know 🧵 https://t.co/gFhsTYIsMv
GSK's New CEO Targets Proven Science, Eyes Obesity Drugs
The FT's report on GSK's $950M deal to buy 35Pharma and its pulmonary arterial hypertension drug includes an interesting unnamed insider's quote that new CEO Luke Miels is going after assets where "the science is relatively settled." Maybe leaning into...
Circulating piRNAs May Predict Elderly Survival and Longevity
Strong claim that needs independent replication: "Our findings provide compelling evidence that circulating smRNAs—especially piRNAs—are powerful predictors of survival in older adults and potential biomarkers of longevity" https://t.co/oi4220zxGY https://t.co/yk3MX4VdQj
Novo's Vivtex Deal Could Unlock Superior Oral Obesity Meds
With Vivtex deal, Novo gains a chance at better oral obesity drugs https://t.co/DhIelbC9jI by @gwendolynawu $NVO #obesity
Dr. Hotez Warns of America's Vaccination Crisis
Stand Up! with Pete Dominick: 1538 Dr Peter Hotez on the state of Vaccination in America. My latest with @PeteDominick https://t.co/LsuvmglNBx
China's Asset Prices Rise, Ending Bargain-Basement Era
Assets from China are ubiquitous but China is no longer the 'bargain basement' in terms of deals. Will be interesting to see how rising prices impact deal interest https://t.co/yM3P28QCDc $XBI $IBB $BBC https://t.co/W61XACPX7O
We All Ingest GMO Carbon From the Atmosphere
Can anything really be “GMO-free” these days? The answer is definitely no. A significant % of all atmospheric CO2 was made by GMO crops in the last 30 yrs, and these molecules cycles back into all plants and into your...
Bull Slams Wedbush's ABVX Report as Terrible
Lot's of requests to discuss the Wedbush report on $ABVX today. It's a doozy. Obviously I'm a biased bull so sure, take my thoughts with a grain of salt, but this is truly, awful work from my perspective....
FDA Says Data, Not Numbers, Prove IBRX Benefit
Th FDA is absolutely correct, and confirms what I’ve said all along. This $IBRX @DrPatrick “lymphopenia” push means nothing until or unless they come up with real data showing patient benefit. The “number” doesn’t matter.
Biotech Embraces Data‑Driven Mornings, Gains Afternoons
I love that biotech has returned to the "data in the morning, raise in the afternoon" way of living its best life. $VIR $PVLA
Former Pfizer CMO Joins $HELP, Credibility Boosted
$HELP adds former Pfizer CMO Dr. Freda Lewis-Hall to its board. Big pharma credibility bump. Now the pipeline has to earn it. https://t.co/CK7PsBE9B8
FDA Needs Clear Standards for Rare Disease Trials
If FDA is serious about moving with urgency in rare disease, the key question is evidentiary standards. Speed matters. But clarity on what constitutes “adequate and well-controlled” in ultra-small populations matters more. Predictability > rhetoric https://t.co/XVXg71NmGq
AI Must Model Dynamic Biology, Not Static Proteins
There is one major barrier blocking AI from truly reshaping biology and no one is talking about it. Biology is not static. Most AI breakthroughs so far have treated proteins, cells, and systems as frozen objects. That abstraction worked to get...
Hepta‑refractory Myeloma Arises via Sequential CD38
The evolution to hepta-refractory myeloma involves sequential loss of CD38, BCMA and GPRC5D [Feb 17, 2026] Riedhammer et al. @LeukemiaJnl https://t.co/EyGBz7sEQz #mmsm #PrecisionMedicine #caxtx
Future Wedbush Take on ABVX M&A Will Be Hilariously Absurd
Looking forward to dusting off some of these absolutely asinine takes from the Wedbush report after $ABVX M&A. It’ll be even funnier then than it is today. Until then, 🥱
Vir Surges After Astellas Partnership and T‑cell Data
Vir climbs on Astellas deal, study results for ‘masked’ T cell engager https://t.co/336c9FJdjt by @Lilah_Alvarado $VIR + 27% $JANX $ALPMY
Novo Slashes GLP‑1 Prices; Palvella Spikes on Data
Novo to cut GLP-1 drug prices; Palvella soars on study data https://t.co/UAs4IETgPX $NVO $PVLA+ 32% $GOSS $VNDA $PFE #biotech
Roctavian Withdrawn, Exposing Science‑to‑market Gap
Failing to find a buyer, BioMarin is voluntarily withdrawing Roctavian from the market. A fitting end for what was once one of the highest-profile gene therapies in development — and a reminder of the gap between promising science and a viable...
Novo Highlights New Data on Triple‑Target Obesity Therapy
Novo, searching for a spark, spotlights new data for three-pronged obesity drug https://t.co/pPo9Oc1wlF $NVO $LLY #obesity
FDA Draft Guidance Boosts Individualized Therapy Innovation
FDA Issues Plausible Mechanism Pathway Draft Guidance to Spur Innovation for Individualized Therapies https://t.co/OZW6Q27zMa via @Inside_PM
