Top Tweets for #baseEditing
Milestone achieved! The first SCD patient treated in China with our high-precision base-editing therapy CS-206 has remained VOC-free for >15 months post-engraftment, achieving the primary efficacy endpoint. No product-related AEs have been observed to date. #SCD #BaseEditing
One infusion of VERVE-102 cut LDL by up to 62% with no serious adverse events. So where does base editing now sit vs CRISPR? We break it down. https://t.co/12CFSiGQdP #BaseEditing #CRISPR #Biotech
Our study shows that the AI-designed nuclease Plant OpenCRISPR-1 (POC1) enables genome knockout, base editing and prime editing in rice, highlighting its promise as a compact next-generation plant genome editing platform
#GenomeEditing #CRISPR #PlantScience #BaseEditing #Rice #AI
$BEAN permanent DNA edit vs. chronic RNA $WVE - more compelling 1time treatment efficacy vs more traditional maintenance treatment. More traction and ESTABLISHING longer term #baseEditing #CRISPR platform application - how much long wait for $40-50, fair val $100
$XBI $IBB $arkG
$WVE also with the AATD update.
For 400mg multi-dose, total serum AAT up to 13.6uM up from 11.9uM for 200mg. Dose response and multi-dose vs single-dose increases modest.
Will be interesting which dose will be chosen: 200mg biweekly or 400mg monthly.
Patients should have 2 options now for AATD. Good. $beam #RNAediting $krro $prqr
https://t.co/z8w1WyDfAZ
Leveraging adenine #BaseEditing, researchers demonstrate high editing efficiency of mutant SCN1A in a newborn mouse model of #DravetSyndrome and show it can lower seizure frequency while boosting survival. @davidrliu @broadinstitute https://t.co/ZyEIdqSxbQ

#geneTherapy #CRISPR #baseEditing #primeEditing #rareDisease is HUGE shirt covering has been sealed over the weekend - $XBI $QURE $RGNX $NTLA $BEAM $CRSP $EDIT $PRME $CRBU $fate $SANA $pacb $DNA $allo $IOVA $LEGN - $NVS $NVO $arkG
New FDA Acting Head of CBER - Karim Mikhail (former AMRN CEO and Exec at MRK , below his comments at JPM 2026 - $QURE $REPL https://t.co/Tfvs25vZWU

Time 2get 1st gen #geneTherapy job DONE, commercialize #CRISPR #baseEditing #primeEditing applications, while focusing on the practical delivery of 2nd generation 🧬 advanced treatments in time for #aging #millennials #genZ
$NTLA $BEAM $EDIT $CRSP $PRME $crbu $rgnx $QURE $arkG
What a night! 🦖 We loved seeing everyone networking alongside the exhibits and celebrating an unforgettable week. It was the perfect way to wind down, toast the incredible momentum of #ASGCT2026, and wrap up our time in Boston.
Thank you to everyone who came out to celebrate with us!

Next #FDA will continue revamp started by @DrMakaryFDA #drMakaryFDA of 1gen #geneTherapy #CRISPR #baseEditing #primeEditing
#AI investors have 2target the same 2expedite next gen treatments that will set stage for more advanced @antiAging and #transformative curable treatments⚕️
@GeneInvesting @RolandoAMoreno Yes, 25% prime editing will likely outperform TRIKAFTA for lung function based on cell data, see the blue vs pink curves.
My guess is that $PRME will push towards >50% editing efficiency for IND-enabling.
An optimized adenine #BaseEditing strategy can repair the “untreatable” 1717-1G>A #CysticFibrosis mutation in cells and organoids with potentially promising efficiency rates and little off-target editing, according to new work. @CIBIOcrispr @UniTrento https://t.co/xfHGGSJLxj

An optimized adenine #BaseEditing strategy can repair the “untreatable” 1717-1G>A #CysticFibrosis mutation in cells and organoids with potentially promising efficiency rates and little off-target editing, according to new work. @CIBIOcrispr @UniTrento https://t.co/xfHGGSJLxj

Congrats to Drs. Rebecca @AhrensNicklas and @kiranmusunuru - the two leading scientists - together with Prof. @davidrliu, who are responsible for one of the most important breakthroughs in the Gene Therapy and CRISPR Gene Editing field - when a personalised and custom-made genetic Bade Editing treatment helped to cure baby KJ Maldoon’s rare genetic disease🧵👇, on been named to the TIME100 List honoring the 100 most influential people in the world today! A well-deserved recognition!

And #geneTherapy #CRISPR #baseEditing #primeEditing #CARt #Til valuations are only getting catch up with humongous clinical development progress $BEAM $CRSP $NTLA $EDIT $CRBU $LEGN $allo $iova $SRPT $QURE $rgnx - $arkG $arkK $XBI $RUT 🚀📈
This 📊👇 - from Stifel’s @TimOpler excellent and comprehensive BioTech and Pharma market outlook, shows that 2026 is absolutely on fire and is currently on track to generate >$250B (!) in M&A deals alone! If this current pace continues - 2026 will become the second most active year in the history of the BioTech and Pharma ecosystem!! $XBI

A new #Primer from @KomorLab, @KDombrink, @benrpollak on #BaseEditing, a method for precise genome editing through programmable single-base changes. Base editing has application in high-throughput screening, disease modeling, and therapeutics. 🧬https://t.co/ccJl7OeSFt
With #IranWar clearly in negotiating or serious #IranRegime destruction phase, #biotech #geneTherapy #CRISPR #baseEditing #cellTherapy #carT sectors will soar 25-30% to establish #takeover level protection while #bigPharma is focusing on easy targets
$XBI $arkG $GILD $PFE $JNJ
Expect big moves $NTLA $BEAM $CRSP $edit $crbu $PRME $LEGN $allo $RGNX $SRPT $QURE $vir $arkG - $XBI $IBB
@bradloncar @WassimLaroussi3 @BiotechTV Hope #geneEditing #CRISPR #baseEditing leaders will be last: $BEAM fair value (fv) today $60-75 vs $24 now, $NTLA $40-50 vs $13, $CRSP $90-100 vs $49, $EDIT $7-10 vs $2.70, $PRME $7-10 vs $3.30 $CRBU $7-9 vs $1.90, #cellTherapy $LEGN $50-75 vs $19, $allo $10-12 vs $2.5 $arkG $IBB
Watch #geneTherapy #Crispr #baseEditing #PrimeEditing #cellTheraoy
$NTLA $EDIT $BEAM $CRSP $PRME $crbu $allo $regnx $DNA $SRPT $LEGN $VIR
It looks like the White House is finally taking US-China biotech competition seriously. The FDA is putting forward an expedited IND pathway that would shorten time to first-in-human.
Overdue, but good news for American biotech.

This completely unrisks $BEAM and overall #geneTherapy #CRISPR #baseEditing #primeEditing #biotech
🆙 trajectory is cleared $NTLA $CRSP $edit catalysts are next $PRME $crbu $rgnx $SRPT $qure is interesting
$XBI $arkG $LLY
Proud to share these BEACON trial data published today in @NEJM of risto-cel base editing of gamma globin promoters in #SickleCellDisease More hope for our #SickleCellWarrior families. A great team of investigators. https://t.co/Kgb5rbzERe
@DFBC_PedCare @BostonChildrens
#biotech is the way! Sector and it’s leaders are beneficiaries of #AI and #OperationEpicFury panicky market selloff is #OneInLifetimeOpportunity for individual investors.
#geneTherapy #geneEditing #baseEditing #PrimeEditing #carT #TIL $NTLA $BEAM $edit $CRSP $PRME $crbu $allo
From Cowen: "Biotech has been the beneficiary of these concerns as investors rotate into Biotech from Tech and other healthcare subsectors whose business models are more threatened by the bots"

#BaseEditing is still a relatively new #biotechnology. Researchers are working to improve its efficiency, while lowering the incidence of unwanted bystander edits. @KomorLab has developed a new tool to do this. Their work appears in @NatureBiotech. https://t.co/smW2lVLjzn

HUGE #2026 4 #BIOTECT is not a dream -🏧#Pharma takeovers of mid-stage+promising platforms is HUGE 4 #geneEditing #CRISPR #baseEditing #primeEditing #cellTherapy #carT #TIL
#OperationEpicFury meeting key objectives will trigger BIGGEST rally in years 🇺🇸
https://t.co/3La4zBmUrY
I have just spoken with Iranian President Massoud Pezeshkian.
I called on him to put an immediate end to the unacceptable attacks Iran is carrying out against countries in the region, whether directly or through proxies, including in Lebanon and Iraq. I reminded him that France is acting within a strictly defensive framework aimed at protecting its interests, its regional partners, and freedom of navigation, and that it is unacceptable for our country to be targeted.
The unchecked escalation we are witnessing is plunging the entire region into chaos, with major consequences today and for the years to come. The people of Iran, like those across the region, are paying the price.
Only a new political and security framework can ensure peace and security for all. Such a framework must guarantee that Iran never acquires nuclear weapons, while also addressing the threats posed by its ballistic missile programme and its destabilising activities regionally and internationally.
Freedom of navigation in the Strait of Hormuz must be restored as soon as possible.
I also urged the Iranian President to allow Cécile Kohler and Jacques Paris to return safely to France as soon as possible. Their ordeal has gone on for far too long, and they belong with their loved ones.
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