BBC Infographic Explains UniQure Gene Therapy for Huntington
Especially after the recent disappointing regulatory update regarding $QURE AMT-130 clinical candidate for treating Huntington disease and the FDA refusal to move forward 🧵👇- here is an excellent Infograph by the BBC which explains how exactly the mechanism of @uniQure_NV’s Gene Therapy works and how it could potentially treat Huntington's disease. $XBI
Cranberry-Derived Ergostane Restores Ovarian Function in Mice
Ergostane steroid, as one of the major contributor to cranberry derived extracellular vesicle nanoparticles, restores ovarian function of murine premature ovarian failure https://t.co/CFcjDvzMnU https://t.co/le7VTHjEKa
Engineered Brain Cells Clear Dementia Plaques in Mice
Boosted Brain Cells Erase Dementia-Linked Proteins “At nearly six months of age, when untreated mice normally have brains saturated with harmful plaques, brains of treated mice were plaque-free. Meanwhile, older mice with plaque-saturated brains at the time of treatment saw a...
UT San Antonio Awarded $38B ARPA-H Grant for Drug Trials
UT San Antonio has been given a $38biin ARPA-H grant to study the health benefit effects of three drugs in humans, namely, rapamycin, dapagliflozin and semaglutide …🧵 https://t.co/fAX14ngoyD
Ribosome-Targeted Drugs Redefine Psychiatry and Beyond
Ribosomes as Drug Targets. The Quiet Rewrite of Psychiatry and Beyond. | Ep. 950 https://t.co/gKlWpzO1Ci https://t.co/ImGOmUVrSO
Gene Therapy Leaps: Base Editing, CAR‑astrocytes, iPSC Approval
stunning week in cell and gene therapy: in vivo base editing of PCSK9 delivered via LNP, w clinical data (Nat Med), preclinical POC of CAR-astrocytes engineered w AAV (Science) and of self-amplifying RNA delivered IM (also Science), Japan approves gene...
L
Doesn’t look like $LLY and $NVO have much to worry about from $RHHBY obesity 🤷🏻♂️
Self‑amplifying RNA Could Protect Future Heart‑attack Patients
Will we give a shot of protective, self-amplifying RNA in patients with a heart attack in the future? @ScienceMagazine an intriguing innovation https://t.co/FcJme1ElyU https://t.co/lABFRkR3qg
Bladder Cancer Landscape Shifts, Opening New Strategic Opportunities
When cancer niches are rapidly changing they create both a vacuum of indecision and fresh opportunities for smart players. Here we explore bladder cancer and look at where the field is headed... https://t.co/cf93Qb8dhe https://t.co/fx5V1MVwYN
Eli Lilly Courts Employers to Expand Obesity Drug Access
Lilly targets employers in new bid to broaden access to obesity drugs https://t.co/Y6fmcmTcf1 @ByJonGardner $LLY $NVO #obesity
China's Rise Threatens US Gene Therapy Market
China competition is coming for the US's multimillion-dollar gene therapies -- a must-read from @Jared_Whitlock about what's happening there and how it will affect access around the world: https://t.co/F7KaMv3Kkp
Yamanaka Stem Cells Earn Conditional Approval for Heart and Parkinson's
20 years of Yamanaka stem cell factor research culminates in 2 conditional approvals for heart disease and Parkinson's disease in Japan https://t.co/8oxKxXYafu by Shinya Yamanaka @CellStemCell https://t.co/U878IxSfSi @ScienceMagazine https://t.co/HCCPaTEP7e
HMPL‑A580 Confirmed as Anti‑EGFR ADC, Impacts ALXO
I speculated last month that HMPL-A580 was an anti-EGFR ADC https://t.co/XFDtZfWWkH and $HCM just confirmed that this is the case. Relevant for $ALXO
Theravance Biopharma Faces Second Layoff Wave After Phase III Flop
5March: Back in 2021, I wrote about a significant round of layoffs at Theravance Biopharma ( $TBPH). The company is cutting jobs again, and has begun a strategic review, after a Phase III failure. Details in my latest for @GENbio:...
Red Blood Cell–PD1 Conjugates Show Promise in Resistant Tumors
Erythrocyte–anti-PD1 conjugates in persons with advanced solid tumors resistant to anti-PD1/PDL1: preclinical characterization and results of a phase 1 trial https://t.co/1GQMlUHn4Y https://t.co/0it5nTvUm0
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's Extremist Shifts Harm Rare Disease Stakeholders
In this week's Biotech Scorecard newsletter: The extremism of the FDA’s Peter Marks and Vinay Prasad has come with costs Two regulators, two extreme regulatory philosophies, one replacing the other. The rare disease community is suffering whiplash. Drugmakers ( $QURE...
GLP‑1 Drugs Cut Substance‑abuse Risk in Veterans
Best evidence yet that GLP-1 drugs reduce the risk of substance abuse, from >600,00 US Veterans across alcohol, nicotine, opioid, cocaine, cannabis @bmj_latest @zalaly @Miao_Cai_SLU https://t.co/rcqUt0kY77
Cell‑free DNA Test Predicts Liver Disease and Mortality
We've used a blood test—cell free DNA—for detecting cancer or prenatal fetal abnormalities. It turns out it can be used to detect liver diseases and all-cause mortality from other conditions @ScienceTM https://t.co/IJxPMq7hGU
FDA COO Butler Retires After One Year; Deputy Assumes Role
after just one year in the position, FDA COO Butler will retire, and his deputy will take over in April - https://t.co/9qmmIiuWX2
Blackstone Invests $400M in Teva, Sanofi Gut Drug
Blackstone puts $400M into Teva, Sanofi gut disease drug https://t.co/CkxPaI6CPr @ByJonGardner $TEVA $SNY $MRK $RHHBY
CTMX Soars 660% Ahead of Masked ADC Catalyst
$CTMX, +660% in 12 months, approaches its masked ADC catalyst. Via @ApexOnco -> https://t.co/oQI1iQiV4N ( $JANX -58% in the same timeframe)
Autonomous Labs vs Traditional Automation: Key Differences Explained
I get asked a lot how Autonomous Labs are different than traditional lab automation, here’s how 👇 Happy to take Qs in comments too https://t.co/AdGMiQWHHT
Future Cancer Blood Tests May Accurately Screen High‑Risk Groups
Cancer blood tests for screening? Not the way they've been studied to date (age 50+) A new feature @Nature “I’m confident we’re going to see more accurate tests going forward. In high-risk groups, such as those with a genetic predisposition,...
Moderna Won’t Risk a Public Jury Verdict Today
If you were Moderna, would you have gambled going in front of a jury made up of the general public these days? I don’t think so.
Tirzepatide Delivers up to 21% Weight Loss, Dose‑dependent
Tirzepatide: In the SURMOUNT-1 trial, weight reduction percentages were dose-dependent: 5 mg: ~15.0% weight reduction. 10 mg: ~19.5% weight reduction. 15 mg: ~20.9% weight reduction (Jastreboff et al., 2022). https://www.nejm.org/doi/10.1056/NEJMoa2206038 https://www.gatlan.com/ @GatlanHealth 
Prime Medicine Pursues Approval After Two-Patient Gene Editing Trial
Prime Medicine $PRME to seek approval for gene editing treatment after two-patient trial https://t.co/nBnrUzqSCN via @Jasonmmast
Pierre Fabre Pushes to Revive US Cell Therapy Approval
Pierre Fabre seeks to revive US approval chances for spurned cell therapy https://t.co/gJeklT8asd @ByJonGardner $ATRA $QURE $RGNX
FDA Guidance Boosts Biotech, HHS Calls It Fake
Did the FDA’s individualized therapy guidance put a spike in the heart of a startup biotech? Fake news, says the HHS. Yeah, it did, says an insider. Exclusive from @RLCscienceboss and @Jared_Whitlock https://t.co/IN9o4VIp03
LLM Medical Research Overwhelmingly Lacks Real Clinical Data
A new systematic review of LLMs in medicine @NatureMedicine @ekoermann https://t.co/npvDeqUvel “Despite thousands [4,609] of publications since late 2022, only a small fraction use real clinical data and just 19 randomized trials exist.”
Integrating Omics Data, Not Algorithms, Is Pharma’s Biggest Challenge
The hardest problem in pharma data science isn't the algorithm. It's getting three different omics datasets to agree with each other. That's exactly what we'll dig into at #ddpeast26 on June 10 in Waltham. I'm joining as a panelist and roundtable moderator....
T‑cell Engagers Offer New Autoimmunity Treatment Opportunity
T-cell engagers for autoimmunity. Listen to Ken Song describe the opportunity as CEO of San Diego-based Candid Therapeutics. Sponsored by @AlphaSenseInc & Dash Bio. https://t.co/Ct2viH00c4
APGE Should Drop OX40 Bispec, It's Just a Black Box
$APGE should can the OX40 bispec IMO. You’re basically just tacking on a black box with that MOA at this point…
FDA Doubts QURE Gene Therapy Benefit, Leaks to Media
New reporting from me taking you inside the $QURE - FDA Huntington's gene therapy imbroglio. I spoke to a senior FDA official late yesterday, who told me agency reviewers "are not convinced there’s any therapeutic benefit of the product. If...
AI‑Discovered Gal‑3 Blocker Cuts Inflammation, Boosts Memory in Mice
Alzheimer’s research is increasingly looking beyond plaques and tangles to the brain’s immune response. A study published in Elsevier's Pharmacological Research journal used AI to screen 1.6M compounds for a Galectin-3 (Gal-3) blocker that could cross the brain barrier. In Alzheimer’s-like...
P‑tau 217 Blood Test Outperforms Total P‑tau 217 For
The breakthrough blood test p-tau 217 for risk and diagnosis of Alzheimer's disease is more accurate than total p-tau 217, in a head-to-head comparison using PET imaging https://t.co/faILpVr9Ua https://t.co/lzUgFnFUP5
GLP‑1 Weight‑Loss Drugs Work Better for Women
A systematic RCT analysis of GLP-1 drugs show they are more effective in women than men for weight loss https://t.co/cycwi4nnN5 https://t.co/KOl0FgKaZD
Roche's MS Drug Shows Promise, Approval Still Uncertain
Roche pill succeeds in another MS study, but approval questions linger https://t.co/JY8uZCI5iA @ByJonGardner $RHHBY $SNY
FDA Demands Extra Study for UniQure's Huntington Gene Therapy
UniQure says FDA wants another study of Huntington’s gene therapy https://t.co/1pgFDoE2V9 by @realJacobBell $QURE - 35% #GeneTherapy #Huntingtonsdisease
AI Pathology for Cancer Markers Hindered by Shortcut Learning
AI of whole slide images for cancer molecular markers is not ready for clinical use due to confounding and biases ("shortcut learning"), supported by multiple examples @natBME https://t.co/zG8uVIxrJf https://t.co/hQ0tjiapIJ
Exercise‑derived Muscle Vesicles Boost Brain Microglia, Improve Cognition
A new mechanism for improved cognitive function from exercise in the Alzheimer's disease model Skeletal muscle EC vesicles interact with the brain and rev up microglia function https://t.co/eZu14YVasY
Subq Inventory Expense Shifts Income, Potentially Doubles TAM
Note for $TGTX, this year has $100m in expenses building inventory for Subq - from a theoretical accounting perspective should really be capitalized (and then written-off if failed or amortized in year of sale), but in actuality gets deducted from...
Aardvark Pauses Pivotal Prader‑Willi Trial over Safety
Safety concerns spur Aardvark to halt key Prader-Willi drug trial https://t.co/PWkOuDMIvo $AARD - 52% $SLNO
Vaccine Pioneer Warns: We’re Heading Downhill.
Stanley Plotkin, known as the “godfather of vaccines,” in an interview with @HelenBranswell: “All I can say is that I’m beginning to regret having lived so long — because we’re going downhill.” https://t.co/A2zcoP2Ghd
Antibody Plus Ozempic Drives Significant Weight Loss
New @NatureMedicine A randomized trial of antibody vs activin type II receptors with or without semaglutide (Ozempic). The antibody, bimagrumab, promotes muscle growth. Marked weight loss with the combination https://t.co/XUK93GTAmA
Veteran Vaccine Pioneer Warns of Eroding Confidence
Stanley Plotkin had a major hand in the development of a number of vaccines in use today; he designed the rubella vaccine. He remembers the world before widespread use of vaccines & knows what's coming as vaccine policy is rewritten...
GENB CEO Describes Emotional Whirlwind Going Public
2March: How did @generate_biomed CEO @mike_nally describe his whirlwind of emotions as his company went public? Read what he and CFO Jason Silvers, MD, JD said about $GENB plus updates on $CASIF, $NVAX, $PMVP & $SRPT in my latest StockWatch...
Future: Real-Time Immune Monitoring Like Glucose Tracking
Someday we'll be able to track our immune system like we do glucose Today @NatBME https://t.co/HFcFiUeyFg Previously @ScienceMagazine https://t.co/EcgBdwGxcd https://t.co/SpoZ5bLtjx
Ozempic Reverses Osteoarthritis Cartilage Damage, Study Shows
I used to teach that osteoarthritis was "wear and tear" — lose weight, take painkillers, wait for a knee replacement. A study just published in Cell Metabolism proved that wrong. Semaglutide (Ozempic) didn't just reduce joint pain in osteoarthritis patients — it...