There is 1one truth and 2two sides to every story
Joined May 2015
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RealityB retweeted
Harvard chemist and molecular biologist David Liu invented gene editing tools that can correct and rewrite defective genes. His research could one day help hundreds of millions of people with genetic diseases, including sickle cell and muscular dystrophy. cbsn.ws/4oyLApL
By focusing on individual trials and indications, we might be missing the BIGGER, truly #transformational potential of the underlying #geneTherapy, #geneEditing, and #CRISPR PLATFORMS.
&2021 highs not “bubble”, just 2early $NTLA $CRSP $EDIT $BEAM $PRME $DNA $arkG $XLV $XBI $IBB
RealityB retweeted
Harvard chemist and molecular biologist David Liu invented gene editing tools that can correct and rewrite defective genes. His research could one day help hundreds of millions of people with genetic diseases, including sickle cell and muscular dystrophy. cbsn.ws/4oyLApL
RealityB retweeted
Delusional thinking about healthspan and longevity, now from China
"In 5 to 10 years nobody will get cancer."
"Living to 150 is definitely realistic. In a few years this will be the reality."
@ATHigginsNYT w/ @prof_horvath @gladyshev_lab
nytimes.com/2025/11/08/world…
This episode might weight huge on #geneEditing ##CRISPR #baseEditing #primeEditing - supported by groundbreaking #GeneEditing data reads from $CRSP and $NTLA, the🧬 sector is sets itself up for another winning inning, indicated support from #FDA 👀 at extensible platforms $IBB📈
Intellia released strong data on their one-time CRISPR treatment for HAE (hereditary angioedema — sudden, painful swelling attacks).
31 out of 32 patients had zero attacks and stopped all meds.
This space one of the few where US leads China.
Will FDA become supportive?
$NTLA
Main Street influencers reporting on $CRSP #NTLA #geneEditing #CRISPR news 🗞️as much more forward looking and groundbreaking - $IBB $XBI $arkG 2025 year end ❤️🩹 recovery is picking up steam 📈
🇺🇸 NEW GENE-EDITING DRUG COULD LOWER YOUR CHOLESTEROL FOR LIFE
Scientists just dropped a potential game-changer: a one-time CRISPR gene-editing treatment that slashed cholesterol by about 50% in a new human trial.
15 people got one IV infusion and boom, their cholesterol and triglycerides plummeted without a single daily pill.
It works by knocking out the ANGPTL3 gene in your liver, which normally helps your body crank out cholesterol.
Doctors say it could be a lifetime fix for millions, no more forgetting statins or arguing with your doctor about your numbers every year.
Yes, it’s still early, and yes, it’s expensive, but this could be the first real step toward a permanent solution for heart disease.
If the future of medicine is one-and-done gene editing, it might’ve just arrived.
Source: NPR
RealityB retweeted
Tonight’s episode of @60Minutes will include a segment on the impact of federally funded science and recent cuts to science funding. Our lab’s development of base editing and prime editing and their clinical applications will be among the work described in the episode. paramountpressexpress.com/cb…
#geneTherapy #CRISPR #LDL
via wsj.com/health/pharma/more-d…
#InPlay Monday 11/10 $CRSP $EDIT $NTLA $arkG $beam $prme $IBB $XBI
Original Article: Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 nej.md/43doGvu
@AHAScience | #AHA25
RealityB retweeted
$ntla the tourist shorts may not be old enough to remember that one of the main applications of tech is to go after therapeutically validated targets (e.g. TTR validated by $alny RNAi) and to provide a lifelong alternative.
Intellia is executing on this promise almost* to perfection.
Further benefits: less off-targeting, delivery-related side effects only one time and in pre-defined time window.
*need to be more careful with inclusion criteria and failure to implement risk mitigation earlier
$CRSP “While it is still early, ultimately technologies like this will likely play a role in supplanting every two weeks injections and taking daily 💊”
#geneTherapy #CRISPR pioneers clearly advanced $NTLA $EDIT - shaping a new #curable drug class $IBB
Via wsj.com/health/pharma/more-d…
RealityB retweeted
🚨 Breaking from #AHA25 & @NEJM
The first in vivo CRISPR–Cas9 gene-editing trial targeting ANGPTL3 (CTX310) shows robust lipid reductions after a single infusion:
•⬇️ 80% ANGPTL3,
•⬇️ 49% LDL-C,
•⬇️ 55% triglycerides,
with no dose-limiting toxicity.
A potential once-in-a-lifetime therapy to permanently lower atherogenic lipoproteins.
@society_eas
nejm.org/doi/full/10.1056/NE…
RealityB retweeted
$crsp ANGPTL3 data observations:
1) unlike prior RNAi/ASO, solid LDLc reduction despite overall similar ANGPTL3 reduction; may point toward role of ANGPTL3 in hepatocytes (digital nature of CRISPR vs RNAi gene modulation)
2) relatively high inter-patient variability of response
3) poor dose response with this non GalNac-LNP complicates finding safe/efficacious dose
4) n=15 additional cases in support of NTLA-2001 DILI being target-specific
📢📢📢 $NTLA lonvo-z Phase 1/2 - very positive up-to 3 years follow-up data read :
stocktitan.net/news/NTLA/int…
$NTLA is not going backwards - released datapoints are clearly VERY positive. nex-z NTLA-2001 hold is a serious blow, but still has better changes of resumption. Implicit collaboration and perfect timing pushing the science along with $CRSP & $EDIT #invino
stocktitan.net/news/NTLA/int…
$NTLA is not going backwards - released datapoints are clearly VERY positive. nex-z NTLA-2001 hold is a serious blow, but still has better changes of resumption. Implicit collaboration and perfect timing pushing the science along with $CRSP & $EDIT #invino
stocktitan.net/news/NTLA/int…
#FDA to create a ‘plausible mechanism pathway’ for conditional approval” for platforms of “bespoke therapies.” - #InPlay to benefit #CRISPR pioneers and extensions followers -.
biospace.com/fda/fda-eyes-ac… via $EDIT $NTLA $CRSP $BEAM $PRME $CRBU #arkG $IBB $XBI $XLV
1/🚨WOW! @CRISPRTX has presented today positive Phase 1 data from its ongoing clinical trial evaluating CTX310 - $CRSP In Vivo CRISPR Cas9 Gene Editing therapy which targets ANGPTL3. Today’s data shows that a single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum-89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose. This data set demonstrates the potential of CTX310 to deliver meaningful and sustained lipid lowering following a single-course intravenous (IV) infusion. This also strengthens the potential of $CRSP In Vivo cardiovascular Gene Editing portfolio. $XBI
RealityB retweeted
1/🚨WOW! @CRISPRTX has presented today positive Phase 1 data from its ongoing clinical trial evaluating CTX310 - $CRSP In Vivo CRISPR Cas9 Gene Editing therapy which targets ANGPTL3. Today’s data shows that a single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum-89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose. This data set demonstrates the potential of CTX310 to deliver meaningful and sustained lipid lowering following a single-course intravenous (IV) infusion. This also strengthens the potential of $CRSP In Vivo cardiovascular Gene Editing portfolio. $XBI
Some of the larger population geared #geneTherapy #CRISPR #transformative curable treatments can be expanded if available in 2 flavors: 1&DONE, and lower 2 dose course to limit excessive toxicity effects🧬🩺
$NTLA $CRSP $NTLA #AHA2025 $IBB $XBI $arkG
Merck’s $MRK pill to reduce cholesterol on top of statins matches results from injectables statnews.com/2025/11/08/merc… via @cooney_liz #AHA25
RealityB retweeted
#AHA25 and @NEJM data for CTX310 by @ProfSNicholls . A single in vivo administration safely and durably lowered ANGPTL3, reducing triglycerides and LDL, progress toward applying gene editing to more common diseases. @CRISPRTX
