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New data from Dr. Liu's group. It is the result of more than three years of continuously improving CRISPR-associated transposases. A version, dubbed evoCAST, improves the editing efficiency from less than 0.1% of human cells to between ~10%-30% of cells.
One major hurdle of turning the evoCAST into a therapeutic solution that is able to insert large genes is the size and collective complexity of the seven proteins that comprise the system. But Dr. Liu said they are working on streamlining the system further.
https://www.science.org/doi/10.1126/science.adt5199
Good news seems to be on the long-term horizon. The problem is the time it takes to adequately test dosage. Ten patients isn't going to cut the mustard and get this product into Phase III for several years. I have seen this scenario happen with other companies. Stocks get ruined because the companies are more interested in trying to perfect dosage than to take a positive dosage and navigate it through advanced trials. APTOF is a prime example of that type of history.
Based on the CTX310 data, it looks to have a clean safety profile. There were no SAEs, no grade =3 AEs, and no clinically significant changes in either liver enzymes (ALT, AST and/or bilirubin) or platelets across all dose levels. However, just ten patients across all four dose cohorts were treated. The data shows minimal activity at DL1 and DL2, but meaningful responses at DL3 and DL4. A single patient at the highest dose level achieved the most impressive reductions, indicating they could take this into the phase one(b).
Q1 financial results and positive top-line data from phase one trial of CTX310 https://www.globenewswire.com/news-release/2025/05/06/3075498/0/en/CRISPR-Therapeutics-Provides-First-Quarter-2025-Financial-Results-and-Announces-Positive-Top-Line-Data-from-Phase-1-Clinical-Trial-of-CTX310-Targeting-ANGPTL3.html
CRISPR-X is working on CRISPR-associated transposases, base editors, prime editors, epigenetic editors and novel editing. All have (multiple) advantages over oligonucleotides.
Sold my CRSP shares and BOUGHT MORE WVE WAVE THERAPEUTICS
RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; WVE-007 for the treatment of obesity and other metabolic disorders; WVE-N531, an exon skipping oligonucleotide for the treatment of duchenne muscular dystrophy; and WVE-003, an antisense silencing oligonucleotide for the treatment of Huntington's disease (HD). It also engages in the programs, for discovery stage research of PNPLA3, LDLR, and APOB. The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was incorporated in 2012 and is based in Singapore.
Added a chunk today to my stash average is now 42.00
$CRSP
Nice pre market action!
Very deep pipeline with a mega partner VRTX
$CRSP
Starting to turn the corner now!
Glad I re-loaded the low 40's
$CRSP
They will present at the 43rd Annual J.P. Morgan Healthcare Conference (Jan 14, at 2:15 PM PST).
Neglected ugly duckling. $NTLA, $VERV & $CRBU now do trials outside of the US.... Maybe sentiment change is on the horizon -- see below
"The CBER director also apologized for “mistakes” in how the FDA handled early work for in vivo gene therapies, which “led to offshoring of some of the trials.”"
https://endpts.com/fda-vaccine-chief-peter-marks-pledges-to-stay-as-long-as-hes-welcome/#:~:text=The%20CBER%20director%20also%20apologized,%E2%80%9Cit%20is%20really%20important.%E2%80%9D
CHEAP CHEAP
SRPT and BMS announcing nice CRISPR deals with ARWR and PRME should help this too!
CTX130 data in R/R T-cell lymphomas (peripheral T-cell lymphoma or cutaneous T-cell lymphoma) https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(24)00508-4/abstract
The ORR was 46.2%. Of those treated at DL3 and higher, the ORR was 51.6%.
so so so undervalued and misunderstood i believe...... frankly just cheap
#FDA CBER's Marks apparently apologizing (!) for holding back in vivo #CRISPR development in the US:
— Dirk Haussecker (@RNAiAnalyst) November 22, 2024
h/t @endpts pic.twitter.com/AZi8R30F7Y
They now have the ability to put in eight to nine edits. So it is a single cassette that expresses the CAR, can express a cytokine and has an shRNA-like approach to knock down genes. So there's no theoretical limit as to the number of edits, but there may be marginal utility beyond a certain point.
Hopefully, they look to improve donor selection, cell selection and manufacturing, as all could increase efficacy.
OT: In this Dr. Urnov, PhD, laid out the urgent case for major reform in the regulatory appraisal of clinical therapies involving CRISPR gene therapies https://www.liebertpub.com/doi/10.1089/crispr.2024.0082
CRSP will climb tremendously over the years.
SMMT went from $1.00-30.00+ this year cancer drug superior to Mercks Keytruda
ICCM company being over looked that kills cancers by freezing them 97% non recurrence rate for breast cancer after 5 years and superior data to lung cancer safe cryoblation is done in less than hour.
https://www.prnewswire.com/il/news-releases/icecure-medical-reports-positive-topline-results-from-ice3-cryoablation-breast-cancer-study-achievement-of-96-39-recurrence-free-rate-brings-company-one-step-closer-to-providing-women-a-non-surgical-alternative-to-lumpectomy-302092712.html
https://www.prnewswire.com/il/news-releases/icecure-medical-reports-positive-topline-results-from-ice3-cryoablation-breast-cancer-study-achievement-of-96-39-recurrence-free-rate-brings-company-one-step-closer-to-providing-women-a-non-surgical-alternative-to-lumpectomy-302092712.html
Buy range again thanks a bunch
Buy range again thanks a bunch
Hey Monk, Hopefully you bought some shares on Friday@ Sold at 10% discount!
LC
CRISPR!!!
Crispr added some $120 Vertical debit spreads in 2026
$CRSP
I agree.....solid company$$$$$$$$
It may take some time for CRSP returns to a $200 price tag
The same could be said for hundreds of other stocks that soared during Covid mania
Since then the stock has rallied from low points
With all that money the company has to do incredible things with its biotechnology the stock isn’t going to zero
Strong balance sheet with approximately $2.1 billion in cash, cash equivalents, and marketable securities as of March 31, 2024
https://finance.yahoo.com/news/crispr-therapeutics-provides-business-reports-200000059.html
(OT): A new AI paper from Princeton and Stanford https://www.biorxiv.org/content/10.1101/2024.04.25.591003v2.full
CRISPR-GPT demonstrated a marked improvement in gene-editing experiments, increasing the accuracy of target gene modifications by up to 30% compared to conventional methods. In validation tests, it achieved a specificity rate exceeding 95%, with a significant reduction in off-targets. The system also reduced the time required to design and plan experiments by approximately 40%, streamlining the workflow for researchers.
Business Update and Q1 Financial Results https://uk.finance.yahoo.com/news/crispr-therapeutics-provides-business-reports-200000300.html
Highlights (plus new programs utilising in vivo gene editing) https://finance.yahoo.com/news/crispr-therapeutics-highlights-asgct-oral-110000066.html
https://www.globenewswire.com/news-release/2024/04/22/2867236/0/en/CRISPR-Therapeutics-to-Present-Oral-Presentation-at-the-American-Society-of-Gene-Cell-Therapy-ASGCT-2024-Annual-Meeting.html
EDIT is also pursuing myocilin-related glaucoma https://www.globenewswire.com/news-release/2024/04/22/2867244/0/en/Editas-Medicine-to-Present-Pre-clinical-Data-Demonstrating-Progression-of-in-vivo-Medicines-Pipeline-at-the-American-Society-of-Gene-and-Cell-Therapy-Annual-Meeting.html
Hi MD,
CRSP is now down around 1/3 from its February high. The price is where I usually start to accumulate more shares.
Best wishes,
OAG
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