Wei Zhao
@zhaoweiasu
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Following
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515
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Interested in 🧬 Gene Editing: BE & PE || Science Enthusiast || Not Financial Advice
San Diego, CA
Joined September 2010
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Last Seen Profiles
Pinned Tweet
$PRME CEO: "There are other numbers of technologies out there, people called gene writing. I have to tell you from the Prime side, they look awful like Prime Editing to me. I suspect some of those companies are gonna run into our very, very, very robust patent approaches someday"
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@AdamColb_
To be honest, I find several of your points to be problematic. Your focus on women, wealth, or success as the primary indicators of a man's value is neither noble nor conducive to genuine happiness.
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1. Two papers of prime editing variations were recently published. The 1st one is click editor (CE), which combines a DNA-dependent polymerase with nickase Cas9 and uses ssDNA tethered by HUHe for PBS and RTT, serving the same role as RT and pegRNA in the original prime editor.
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In vivo base editing works in humans!
Congratulations to
@KomorLab
,
@NicoleGaudelli
, and
@davidrliu
for advancing gene editing science and inventing base editing technology!
Great work by both the
@BeamTx
team and the Verve team to make it happen!
@skathire
and
@ambellinger
👏🎉
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1. Some people (
@biotech2k1
, et al.) have asked recently about the fidelity, specificity, or off-target (OT) editing of Prime Editing. Instead of answering these questions in a piecemeal fashion, I try to incorporate all of them here in one thread for comprehensive understanding.
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1. The title of the recently published paper, "Genotoxic Effects of Base and Prime Editing in Human Hematopoietic Stem Cells," is quite catchy and unsettling. However, the few points it covers have largely been extensively studied before.
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It’s a good time to remind everyone of the data from $BEAM: multiplex base editing does not alter T cell karyotype or growth (but multiplex Cas9 editing does)
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The news many $BEAM fans have been waiting for.
BEAM-101: the first patient was dosed and also successfully engrafted in 2023 Q4. 👏🤞
Expansion dosing is expected to initiate in 1H 2024 (after 3 patients in the sentinel cohort are dosed and engrafted) 🙏
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1. There has been confusion about how PASTE relates to Prime Editing (PE) + Integrase approach. And the history of prime editing combined with recombinases. I did some research. Here is what I found.
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Reading "Seven technologies to watch in 2022" creates an illusion that I am reading a Nature Biotechnology review, not a Nature review.
$Beam has been mentioned explicitly in this article not once, but twice, and in two technologies. That's significant.
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1. There have been many questions and confusion about how Tessera's Gene Writing technology relates to David Liu’s lab's Prime Editing. I researched papers, presentations, patent applications, and issued US patents for Gene Writing and Prime Editing. Here is what I found.
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My son was born today at Scripps Memorial Hospital La Jolla. His Chinese name is 赵耀, which means shine, just like what a $Beam of light will do.
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When Nature publishes an article announcing PE7, you definitely sit up and take notice.
Here, a team led by Prof.
@bsadamson
has uncovered more secrets in our cells to unlock the full potential of prime editing. 👏
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$PRME: Keith mentioned in today's conference call that the LNP delivery of Prime Editor to the liver appears to be highly re-doseable. No matter how many times they go back, it continues to perform well. He hopes to share more LNP data toward the end of the year.
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BEAM-101 is now actively recruiting and the 1st patient was enrolled on Aug 30th.
This is the 1st Base Editing clinical trial in the US.
BEAM should announce it officially very soon.
Let's hope everything goes well. 🤞
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Okay gang, let’s see what Gene Writing, PASTE, and RT Editing really are.
Prime Editing?!?
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1. Researchers, led by Feng Zhang, have developed a breakthrough technique that uses a molecular 'syringe' from some viruses and bacteria to deliver potentially therapeutic proteins into human cells.
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What just happened? 👇
Humanity's first-ever in vivo gene correction therapy has reached a patient! 🥳
It's also $BEAM's first in vivo base editing program.
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Beam's
@NicoleGaudelli
work of CBE-T (TadA-based C-to-T base editor) is ingenious, which has finally solved the potentially critical safety issue of utilizing CBE in the clinical settings: minimizing gRNA-independent mutation without lowering on-target editing efficiency. 👏🥳
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Good news for $BEAM! According to , Beam's 2nd wholly owned clinical trial, BEAM-201, might have already started around May 25, 2023. It's a Phase 1/2 study aiming to enroll 102 patients, including a pediatric cohort in Phase 1.
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As Beam recently has revealed more details of its future development plan for sickle cell disease (SCD), it is a good time to review the current status and predict the future landscape of treating SCD with CTX001 using CRISPR/Cas9 and BEAM-101/102 using ABE.
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1. Beam provided updates on AATD program. AATD is most commonly caused by point mutation E342K, or PiZ. Misfolded Z-AAT proteins aggregate in liver cells, leading to fibrosis and/or cirrhosis. On the other hand, deficient anti-elastase function leads to lung damage and emphysema.
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$PRME has achieved ~80% of hepatocytes (or ~50% whole liver editing) precise prime editing in vivo in humanized mice, with fidelity ratio >200.
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Why can gene editing therapy make a huge difference?
"It's like living with a new body again really because... the options are unlimited now."
Knox felt ecstatic to no longer be reliant on medication - which she was not using for the first time in 35 years
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$BEAM and $KYMR have long become my favorite biotech companies. Both companies are innovative, ambitious, platform-based, with differentiated pipelines, and pioneers in their field. Now,
@JMaraganore
has joined the board of directors of both companies. What a coincidence!
@KymeraTx
is honored to announce the appointment of
@JMaraganore
to its Board of Directors. Dr. Maraganore joins the Board after having led Alnylam as founding CEO and Director and transformed the field of RNAi therapeutics for nearly 20 years. Learn more:
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1. The 1st $PRME corporate presentation:
Exciting advancements across all fronts!
➡️New preclinical data in FA and CF
➡️No detected OT
➡️New LNP and AAV mice data
➡️PASSIGE
➡️1st DC Q1 23.
➡️pegRNA patent expected Q1 23
➡️strategic collaboration coming
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$PRME. The first ever prime editing trial is on the way! Very impressive and exciting! Congratulations to Andrew, David and the Prime Medicine team! Best wishes for the next step: bringing a cure to the patients. 🎉🙏
We are pleased to announce that
#FDA
has cleared our IND application for PM359 for the treatment of chronic granulomatous disease (CGD), enabling Prime to initiate its Phase 1/2 clinical trial in the US. Details: $PRME
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Really excited about the prospect of using Beam's mRNA/LNP delivery platform to target the liver, muscle, CNS. Just think about those Progeria kids, the desperate father who wanted to save his child with Menkes syndrome, and all the families having Alzheimer's disease patients.
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The FDA has cleared the IND application for $BEAM-301.
It will be the US's first ever in vivo base editing, or precise gene correction clinical trial.
The patient dosing is expected to start in early 2025.
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For anyone who wants to know why base editing and prime editing are revolutionary and how they actually worked and were developed over time, it is a good time to watch or rewatch the video that is presented by David Liu himself.
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Nothing is more exciting than the proposed IPO of Prime Medicine!
Congratulations to Andrew,
@davidrliu
, and many others!
A great moment for many patients suffering from genetic diseases, a new hope is on the horizon!
Also fantastic news for all Beam's shareholders.
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Today marks a historic moment as 1st approval of CRISPR therapies. Congrats to all the scientific pioneers who made this possible: Ishino, Mojica, Horvath, Barrangou, van der Oost, Brouns, Marraffini, Sontheimer, Moineau, Charpentier, Doudna, Šikšnys, Zhang, Church and many more.
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$BEAM will have 4 clinical programs targeting four different diseases this year and will present clinical data for two of them in 2H 2024. The 5th one (ESCAPE) is potentially ready in 2025. It's also possible for Pfizer (6th) and Apellis (7th) to be ready for the clinic in 2025.
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1. Beam's ASGCT23 slides for Stargardt Disease ABCA4 G1961E correction (autosomal recessive inheritance).
The dual AAV ABE has achieved up to 90% A to G conversion in WT iPSC-derived RPE for the A8G Wobble Base and ~10% in the human retinal organoid.
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1. $PRME hits a major milestone: its 1st prime editing data in NHPs achieved up to 50% (average ~41%) whole liver precise editing of p.L348 (a surrogate pegRNA for C->G) in NHPs at day 14 with minimal on-target unintended edits.
Congrats to the $PRME team, Andrew, and
@davidrliu
!
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Really impressed by Beam's two new programs: ESCAPE-1/2 (HPFH/MKSR+CD117) to treat SCD. It has the potential to not only get rid of genotoxic conditioning but also increase editing efficiency indirectly by removing un-multi-plex-edited cells for both ex vivo and in vivo delivery.
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1. It's a total surprise to see OTQ923 from Intellia/Novartis has only achieved 16-22% HbF in 2 SCD patients.
I wonder what went wrong. So I did some research, below are some of my thoughts on this.
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Wow! PE6 has officially arrived! And hold on a second, it's not just PE6 – it's PE6a, b, c, d, e, f, and g, a whopping seven variations tailored for diverse applications! This is yet another significant stride toward therapeutic uses. Big cheers to Liu's lab! 🥳🎉
Today we report in
@CellCellPress
a suite of engineered and PACE-evolved prime editor variants PE6a-g. These new prime editors improve the activity, in vivo deliverability, and in vivo editing efficiency of prime editing.
PDF:
(1/15)
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Ark Invest 2023 Big Ideas features Base Editing, Prime Editing and Targeted Protein Degradation. I like these new modalities as well, and own the stocks of the very best of them: $BEAM, $PRME, $KYMR. RNAi is good but not new. RNA editing is new but I don’t like it.
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I highly recommend
@sawisner
's book, "Building Backwards to Biotech", to anyone interested in the intersection of life science and business, especially those aspiring to become biotech venture capitalists.
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The more I think about Beam's ESCAPE1/2 programs, the more I found them impressive and promising. Or maybe I am too optimistic and went too far😅? Below are my thoughts on these programs, feedback is warmly welcomed.
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1. Prime Medicine $PRME presents preclinical data of PM359 for treating CGD
1⃣Prime Editing precisely corrected the CGD causative mutation in greater than 75% of CGD patient CD34+ cells
2⃣>80% Prime Edited CGD Patient HSC clones
from bone marrow have ≧1 Prime Edited allele
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It appears that BEAM-101 is making good progress. Beam has now opened recruitment at least at 13 sites. It's reasonable to assume that the sentinel cohort is doing well up to this point, and there are already or soon will be at least 10 patients enrolled in the expansion cohort.
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$BEAM has updated its pipeline chart. Progress has been made across multiple programs. 🤞
BEAM-101 in Phase 1/2
BEAM-102 refocusing on ESCAPE and in vivo delivery
ESCAPE in lead optimization
BEAM-301 in IND enabling
BEAM-302 nominated
Stargardt G1961E in lead optimization
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Really good initial response data from $CRBU's CB-010. The key takeaway is that PD-1 KO dramatically improves CAR-T antitumor activity. Whether B2M-HLA-E will improve durability will be answered by CB-011. Unfortunately for CRBU, the two types of edits are not in the same program
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1. Today $PRME's update from CEO Keith:
"There's been an extraordinary amount of interest from a number of pharma companies (to partner with $PRME on prime editing/PASSIGE-enabled CAR-T programs for both oncology and immunology)"
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Tomorrow's the day we can finally say goodbye to the statement that base editing has no human data. Earlier CD-7 CAR-T data is great, but that’s a somewhat indirect data. It’s 7 years since the invention of CBE; 6 years since the invention of ABE. Fast advancement of science!🤞🙏
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Exciting news from Nvelop Therapeutics!
David Liu and Keith Joung have teamed up once again, this time bringing together two viral-like particle (VLP) delivery platforms.
While we're familiar with Liu's lab pioneering the use of eVLP for delivering BE and PE, Joung's lab
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A big milestone for $BEAM today!
Its corporate deck has already been updated: "BEAM-302, CTA cleared in the UK" ✅
@NicoleGaudelli
@davidrliu
@john_evans3
🎉👏
3 anticipated catalysts in Q2 24'
1⃣101: initiate expansion dosing
2⃣302: dose 1st patient
3⃣301: US IND submission
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Great news! All three patients in the sentinel cohort of BEAM-101 have successfully undergone engraftment, and $BEAM should now have data for 5, 3, and 1 months. We can anticipate BEAM to dose approximately 15 patients by February 2025.
Excited to share that we have now completed dosing and successful engraftment of all three sentinel patients in the BEAM-101 BEACON trial for sickle cell disease.
Expansion dosing will now proceed, with the next dose occurring shortly.
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Dr. Kiran Musunuru's pioneering POC work using both base editing and prime editing to treat PKU in mouse models is truly impressive!
When I started working on
#CRISPR
to fight heart disease >10 years ago, I never imagined it might lead to cures for rare inborn errors of metabolism.
Here’s how it’s happening!
Presented/published today
#ASHG23
#ASHG2023
@GeneticsSociety
plenary +
@AJHGNews
+
@HGGAdvances
🧵
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On 05/09/23, the USPTO granted $PRME the foundational and broad covering pegRNA patent, which reflects the essence of prime editing - all prime editing applications will rely on it.
No argument left on who invented prime editing - no more denying and rebranding game, please!
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1. The gene editing company Metagenomi has filed an S-1. Unlike other public CRISPR companies, Megagenomi lacks key foundational patents. They assert that, in contrast to other companies bound by "narrow terms of license agreements," they "are not subjected to these constraints"
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Accelerated approval will be "the norm" for gene therapies, FDA's Peter Marks says.
IMO, compared to traditional gene therapies, gene editing therapies are expected to have more distinct biomarkers and efficacy signals. $PRME's CGD is a great example.
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1. EDIT-101’s clinical results are disappointing: even though 2/2 homozygous patients are responders, only 1/5 heterozygous patients with high-dose may respond. Though it is saddening, we can learn a lot when things didn't work out as expected.
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CR at 6 months! Let's pray it can last much longer!
Alyssa, a 13-year old T-cell leukemia (ALL) patient in the UK, was the first person to receive a base editing therapeutic—T cells with three base edits—and the first patient reported to benefit from base editing: complete remission, 6 months later.
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3K
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Prime Editing Myths Debunked Today:
1⃣Its editing efficiency is low: achieves up to 83% in vivo hepatocyte prime editing
2⃣Its indel/error rate is high: minimal unintended edits (~0%)
3⃣It's too large to be delivered: successfully delivered to NHPs 🐒
Bonus💡: Redosable in NHPs
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1. 4 milestone Prime Editing papers were published
2019: seminal (foundational IP)
2021: epegRNA
2021: PE4/5/max
2021: Dual Flap (TwinPE)
Since then, additional technical advancements include
1⃣ Novel RTs
2⃣ New pegRNA enhancements
3⃣ Synergy with site-specific recombinase (SSR)
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Peter from FDA made some key points:
1. Base editing shows great promise.
2. The slow progress is likely due to regulatory challenges, so simply blaming the management and founders might not be fair or constructive.
3. The FDA is taking steps to expedite the process.🤞
Peter Marks says base editing could be an 'incredible game changer.' But he also conceded there's been a backslide of new therapeutic advancements using gene editing, likely due to discouragement in the regulatory process. More on that dichotomy:
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$PRME has also made good preparations to launch its Prime-0101 study as soon as the IND/CTA is submitted and approved. "We have completed our preliminary clinical trial design and selected leading CGD transplant centers across the US and other countries as clinical trial sites to
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Some quick updates from $BEAM's CEO:
1. BEAM-101 is progressing well, with initial results aligning with expectations👏
2. BEAM believes the LNP toxicity in Verve-101 is due to LNP, not ABE
3. BEAM-302 is expected to be dosed < 1 month, with data anticipated in early to mid-2025
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The beautiful AAV-mediated Prime Editing mice data presented by David Liu today.
Part of this data was already in $PRME’s S-1.
PE3max + epegRNA achieved high editing efficiency in vivo.
The indel rates are nearly zero!
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The FDA is currently investigating the potential risk of developing secondary malignancies with the integration of vectors, such as lentiviral/retroviral vectors, in CAR-T therapies. If this turns out to be true, the PASSIGE + multiplex PE system could prove to be highly valuable
6. PASSIGE
➡️One-step non-viral approach for precise introduction of gene sized cargo into the genome
➡️Integration of genetic cargo at single targeted site in 60% of T cells (Wow!+1 Enabling one-step, non-viral, multiplex PE CAR-T)
Note: rebranded as PASTE (Tome has no license)
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Wow, this work is a remarkable fusion of Liu lab's most potent methodologies: PACE, prime editing, and one of my personal favorite subjects, targeted protein degradation using molecular glues, all spearheaded by Jaron Mercer, a colleague and close friend of Andrew Anzalone!
Today in
@Science_Magazine
we report the laboratory evolution of compact degrons that enable targeted protein degradation triggered by an otherwise-inert small molecule, a multidisciplinary study that integrates organic chemistry, molecular glues, protein evolution, genome
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$PRME's IP position is extremely robust. Not only does it have the foundational prime editing patents, but it also has patents for numerous improvements and variants (PASSIGE, split PE, pegRNA configuration, dual flap and dual guide, DNA-dependent DNA polymerase, and more)
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With its core gene-editing technology, wide-reaching delivery modality, top-notch manufacturing facility, streamlined automation, innovative scientists, ambitious management, and venture connections, BEAM has emerged as one of the central hubs for cutting-edge biotech innovations
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The Cold Spring Harbor Laboratory (CSHL) conference is the world's premier gene editing event. Upon reviewing this year's talks and posters, a clear trend emerges: we're witnessing the era of base editing and prime editing, particularly in the realm of therapeutic applications.🤞
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1. Nov 16 Jefferies, $PRME's CSO: "There are more programs here than we can possibly develop on our own. We're very actively talking with large pharma, medium-sized pharma, and even smaller companies about partnering programs."
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Congratulations to both $BEAM and $VERV! BEAM not only secured $250M, but perhaps even more importantly, it won't need to dip into its cash reserves for co-development in the coming years. VERV now has a good partner, and its 101 data is expected to be promising.
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The first patient in the US being treated by base editing: the beginning of a new era -- precise gene editing without double-stranded breaks. 🤞
Congratulations to all the inventors and individuals who contributed to achieving this significant milestone!
Excited to announce that in August we dosed our first patient in the BEAM-201 trial!
This is the first patient treated for Beam and the first patient in the US treated with a base editing therapeutic.
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The debate on Prime Editing becomes painful not because PE has any major issues but because very few people have actually carefully studied and understood all major PE papers.
The paper below is particularly useful if you do want to learn more about PE.
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1. One of the key arguments against Prime Editor is its relatively large size compared to Base Editor or Nuclease
PE: 6.4kb (4.2kb SpCas9 + 2.2kb MMLV RT) + pegRNA, ngRNA and regulatory sequence (if for AAV) = ~7kb
ABE8: 4.2kb SpCas9 + 0.6kb TadA + sgRNA and regulatory sequence
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$PRME's pipeline at JPM24
1⃣It's still growing (19+)
2⃣Four pillars (core)
Hematology & immunology: 3
Liver: 3
Ocular: 3
Neuromuscular: 7
3⃣Collaborate/license now
➡️PASSIGE CAR-T
➡️CF
➡️Ear: 2
➡️Cardiovascular/cardiometabolic: ?
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1. Aera Tx is launching today.
Very impressive science potential and board members! 👏
"endogenous human proteins...derived from retroelements that self-assemble to form capsid-like structures and which can package and transfer nucleic acid cargo"
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Well, there are no guarantees, but there's a chance $PRME might dose the 1st patient by the end of this year. Should this milestone be achieved within this time frame, it would undoubtedly be a cause for celebration! 🙏🤞
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The FDA aims to implement the "Platform Technology Designation Program for Drug Development."
It is a very positive sign and endorsement for $PRME that Peter Marks from FDA often cites prime editing as an example of this modular platform approach.
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Prime Medicine will IPO on 10/20/22, offering 8.9 million shares at $16~18 with a market cap of $1.5~1.7B.
As Prime is much more valuable than all the other gene-editing companies combined, this is a great bargain for me and I will have to sell my other holdings to buy it at IPO.
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The PE-eVLP work is a bio-engineering masterpiece! 🧬 Despite initial challenges, including minimal editing efficiency (much lower than BE-eVLP) Liu's lab identified crucial bottlenecks and differences. The result: PE-eVLP now has comparable editing efficiency levels as BE-eVLP.
Today we report in
@NatureBiotech
an engineered virus-like particle system for in vivo delivery of prime editor protein-RNA complexes. This PE-eVLP platform can support therapeutic prime editing in animal models of genetic disease.
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1. $PRME published its recent advances in Prime Medicine’s lipid nanoparticle (LNP) and all-RNA delivery platforms for liver disease on Oct 24, 2023.
Expect more updates on this topic on Oct 27th for the 1st NHP data, so It's a good time to start getting in the loop!
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This is a very well-written review paper on LNP delivery for gene editing.
It has identified the most effective formulations of lipid nanoparticles for passive targeting of specific organs, such as muscles, brain, lungs, liver, heart, spleen, and bones.
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Extraordinary statements are concealed within this interview with David Liu.
Question: What recent or ongoing development are you most excited about in your field?
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Key milestones for Base Editing and Prime Editing:
04/20/16: CBE Paper
11/23/17: ABE Paper
10/21/19: PE Paper
02/05/20: Beam IPO
11/08/21: BEAM-101 IND
04/19/22: TvT CAR7 P1
07/05/22: VERVE-101 P1
10/20/22 est: Prime IPO
Very fast progress in the past 6 years!
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The new PE4, PE5 prime editing systems have achieved amazing improvements in editing efficiency and indel reduction in non-HEK293T cells. Below are some of my thoughts.
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BEAM-302: filed CTA to UK (presumably at the end of 2023, so ahead of schedule)
A major milestone for humans as well:
"Opportunity to achieve first ever clinical proof-of-concept of in vivo base editing leading to correction of a disease-causal mutation" 👏🎉
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Prime Editing is the ONLY technology that can potentially make any edit, in any cell - and only make the specific edit.
Therefore, any other technologies claiming similar capabilities are merely imitating Prime Editing.
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One of the possible reasons that Beam found Orbital Therapeutics is that circular RNAs are more compact than their linear mRNA counterparts. As a result, circular RNAs are easier to pack into, and thus more compatible with a broader set of lipid formulation options for delivery.
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The BEAM-VERV-LLY deal is positive for PRME as well. It not only hints for potential PRME deals in the near future, but a robust BEAM also reflects positively on PRME since they are closely connected as sister companies and may collaborate on program development down the road.
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New in vivo PoC data of using CBE to inactivate HBV cccDNA in HBV minicircle mouse model from Beam!
Overall speaking, the data looks good and exceeds my expectations.
A few thoughts/observations as below:
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"Mammoth is also pursuing trickier editing techniques, like base editing, gene writing, and epigenetic editing, what some call CRISPR 2.0 or “CRISPR plus.”"
Mammoth, please be honest and straightforward, what is "gene writing"?
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Good news and bad news for BEAM.
Good ones: all four undisclosed programs (Apellis and Pfizer) have advanced to lead optimization! 🍻
Bad ones: BEAM-102 and Stargardt G1961E were dropped! 💀
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Though I am not an $EDIT shareholder, it saddens me to watch the turmoil at EDIT. To me, the best exit strategy for EDIT is 1. scratch most of its existing pipeline; 2. merge with Beam. Potential issue: Beam does not need EDIT as much as EDIT needs Beam.
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Many thoughts as I go through Prime's S-1.
Overall speaking, great progress has been made in prime editing technology since its 2019 seminal paper. The editing efficiencies are a lot higher in several primary cell types. Well done, Andrew, David, and the prime editing team!
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The SEND system utilizes a human protein, called PEG10, to package mRNA.
PEG10 is a retrovirus-like protein, expressed by a gene that integrated itself into the human genome through ancestral viral infection. So it may not trigger immune responses and thus can be redosed.
NEW: CRISPR pioneer Feng Zhang is building a secretive new startup to solve gene-editing's biggest problem — getting into more parts of the body
Meet Aera Therapeutics, which has raised $200 million to advance Zhang's work:
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Hidden within VERVE-101's setback lies the impressive efficacy of base editing: the initial five participants in the 0.45mg/kg cohort experienced time-averaged LDL-C reductions averaging 46%, surpassing the potency of Inclisiran.
This efficacy remains significant within the
Bad news for VERVE-101: the 6th patient in the 0.45mg/kg cohort experienced Gr3 transient ALT elevation and Gr3 thrombocytopenia.
➡️The Acuitas ionizable lipid is likely responsible for the ALT elevation, and it is presumed to potentially contribute to thrombocytopenia as well
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$PRME CGD program will take >=6 months to dose 1st patient. It is designed for rapid progression, hoping to dose just 2 adults before moving to a pivotal trial for adults, followed by pivotal trials for adolescents and children. PRME hopes to seek approval with <20 patients.
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1. One thing that I cannot emphasize enough is that Prime Editing is currently the only known way that has the capability to efficiently repair transversion point, deletion, insertion, and duplication mutations for both in vivo and ex vivo therapeutic applications.
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If a single copy of the CCR5 mutation can potentially eliminate HIV in some individuals, then gene editing of HSCs should also cure AIDS. Furthermore, utilizing multiplex base or prime editing to treat diseases like SCD or CGD while also addressing HIV could be even cooler.
A 60-year-old man in Germany has become at least the seventh person with HIV to be announced free of the virus after receiving a stem-cell transplant
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FDA's Operation Warp Speed for rare diseases couldn't have come at a better time. $BEAM, $PRME, and importantly, many patients can benefit greatly from this. Plus, Peter Marks has given base editing a thumbs-up before. This might just save the day. 🤞
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A tragedy of brave souls😰💔
1. Terry was too old (27) to qualify for other similar trials
2. First epigenome editing test in human
3. First custom-made
4. Turn on a mysteriously dormant backup copy of the dystrophin gene
5. High-dose AAV can cause deaths
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By now there are ~860 Prime Editing papers published, and 851 of them are not from David Liu's lab.
Thus, Prime Editing has already been under scrutiny from the scientific community since its debut, and no major risk of PE has been identified so far.
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