Editas Medicine Inc (EDIT) オプション

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

From the Q&A's, there was no date given on when they will file an IND. They did not answer if other HSC's are targeted. They did not answer what the LNP targeting moiety is. They did not answer how long they will continue to put money into Reni-cel. They did not answer what doses were given. They also said that redosing is being explored (to achieve higher editing efficiency).

I doubt there will be a partner for Reni-cel unless they get a huge part of in vivo as well.

An update https://www.biospace.com/press-releases/editas-medicine-to-host-strategic-update-webinar-to-detail-progress-towards-2024-goals-including-achievement-of-establishing-in-vivo-preclinical-proof-of-concept

https://www.globenewswire.com/news-release/2024/10/21/2966518/0/en/Editas-Medicine-and-Genevant-Sciences-to-Collaborate-to-Develop-Novel-mRNA-LNP-Gene-Editing-Therapeutics.html

EDIT new 52 week low

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Looking at HbF levels, EDIT-301 appears to achieve slightly higher levels than Casgevy. For total Hb levels, EDIT-301 seems to outperform Casgevy https://finance.yahoo.com/news/editas-medicine-announces-safety-efficacy-070000921.html

However, the ongoing trials and launch are going to be costly, and they aren't working on busulfan free conditioning.

EDIT under $10

(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

Dr. Liu moved on with base and prime editing and just left the company to die on the vine.

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Could be upcoming https://annualmeeting.asgct.org/program/agenda-details?agendaId=25899

If so, CRSP and NTLA have already disclosed preclinical data for in vivo editing.

Does this have any FDA approvals?

No. Plus they are currently focusing on TDT and SCD, both early stage, and both indications becoming more crowed. Also, CRSP should get FDA and EMA approvals for exa-cel in those indications and should have at least five years head start over others.

Will Precision Biosciences be this high or higher?

If the clinical data they present in April/May is good, I expect the SP will increase, but not as high as this. They (DTIL) will also have an upcoming R&D Day (focusing on in vivo programs) mid-year.

How do the two compare?

There are a number of differences. One example, DTIL have yet to show they can do in vivo editing in humans. Another, both have broad plans for in vivo programs, but delivery outside the liver (using current AAVs for many indications) can be problematic.

Does this have any FDA approvals? Will Precision Biosciences be this high or higher? How do the two compare?

PR https://www.globenewswire.com/news-release/2022/05/18/2445810/0/en/Editas-Medicine-Presents-Data-on-SLEEK-Gene-Editing-Technology-at-the-American-Society-of-Gene-and-Cell-Therapy-Annual-Meeting.html

Posters https://www.editasmedicine.com/wp-content/uploads/2022/05/ASGCT2022-EDIT-103-Liu-Presentation.pdf

https://www.editasmedicine.com/wp-content/uploads/2022/05/ASGCT-2022-SLEEK-Zuris.pdf

https://www.editasmedicine.com/wp-content/uploads/2022/05/ASGCT-Kahn-EDIT202-Poster.pdf

https://www.editasmedicine.com/wp-content/uploads/2022/05/ASGCT-Duke-EDIT-101-Immunogenicity-Oral-Presentation.pdf

1106: EDIT-202, a Multiplexed CRISPR-Cas12a Gene-Edited iPSC-Derived NK Cell Therapy Has Prolonged Persistence, Promotes High Cytotoxicity, and Enhances In Vivo Tumor Killing https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=2005

831: SLEEK: A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next-generation Cell-based Medicines https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=1751

ASGCT title:

EDIT-202, a Multiplexed CRISPR-Cas12a Gene-Edited iPSC-Derived NK Cell Therapy Has Prolonged Persistence, Promotes High Cytotoxicity, and Enhances In Vivo Tumor Killing.

PR https://www.bloomberg.com/press-releases/2022-04-08/editas-medicine-reports-engineered-ink-cell-preclinical-data-at-american-association-for-cancer-research-annual-meeting

Poster https://www.editasmedicine.com/wp-content/uploads/2022/04/AACR_2022_Poster_FINAL.pdf

AACR abstract

AsCas12a gene-edited iPSC-derived NK cells constitutively expressing CD16 and membrane-bound IL-15 demonstrate prolonged persistence and robust anti-tumor activities in a solid tumor mouse model https://www.abstractsonline.com/pp8/#!/10517/presentation/16690

ASH PR https://www.globenewswire.com/news-release/2021/12/12/2350352/0/en/Editas-Medicine-Reports-Preclinical-Data-Demonstrating-Robust-Tumor-Reduction-and-Clearance-Using-Novel-Engineered-iNK-Cells-at-the-American-Society-of-Hematology-Annual-Meeting.html

Poster https://www.editasmedicine.com/wp-content/uploads/2021/12/ASH_2021_iNK_poster_presentation.pdf

From the PR: In these experiments, iPSCs were edited using the Company’s SLEEK gene editing technology at the GAPDH locus with a proprietary, Editas-engineered AsCas12a nuclease to knock-in high-affinity CD16 and membrane bound IL-15. iPSC clones were then differentiated into iNKs that were confirmed to express high levels of CD16 and IL-15. Increasing NK cell CD16 expression can improve anti-tumour activity when combined with antibody-dependent cell-mediated cytotoxicity (ADCC)-enabling antibodies. IL-15 is important for NK cell survival, and increasing IL-15 expression prolongs the persistence of NK cells. Knock-in of IL-15 may also eliminate the need to administer cytokines systemically, which can cause severe toxicity.

Results demonstrated that the edited iNK cells exhibited enhanced serial tumour cell killing through ADCC in a2D assay against SKOV-3 ovarian cancer cells and in a 3D tumour spheroid killing assay. The edited iNK cells were also able to persist for a dramatically longer period of time relative to unedited iNK cells. Together, these data provide strong support for the continued development of engineered iPSC derived iNK cells as a potential novel class of therapeutics targeting solid tumours.

''In this promising new research, we demonstrate the use of our proprietary SLEEK technology to knock-in both CD16 and IL-15 into iNK cells. The engineered cells demonstrated potent anti-tumour activity and substantially increased persistence without systemic cytokines, an important limitation with many existing NK cell approaches. We also believe this to be a potentially safer and more reliable approach to developing next generation NK cell therapy medicines because through our iPSC development process, we only select cell clones that have exactly the desired on-target edits, thereby avoiding the possibility of cell abnormalities being introduced,'' said Mark S. Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. ''NK cells are great candidates for off-the-shelf immunotherapy medicines given their high tumour killing capacity and their low propensity for graft-versus-host disease, and we believe these data provide evidence for the potential of future experimental medicines from our iNK program to exert enhanced anti-tumour activity in the clinic in the treatment of solid tumours.''

SITC poster https://www.editasmedicine.com/wp-content/uploads/2021/11/SITC_2021_CD16mbIL-15_KI_iNKs_poster_FINALv.pdf

ASH abstracts

Deletion of CISH and TGFßR2 in iPSC-Derived NK Cells Promotes High Cytotoxicity and Enhances In Vivo Tumor Killing https://ash.confex.com/ash/2021/webprogram/Paper150731.html

Preclinical Development of EDIT301, an Autologous Cell Therapy Comprising AsCas12a-RNP Modified Mobilized Peripheral Blood-CD34+ Cells for the Potential Treatment of Transfusion Dependent Beta Thalassemia https://ash.confex.com/ash/2021/webprogram/Paper149956.html

GRTS and EDIT Biotech Stock Plays Update | Big Events Coming Up!

I chat about some recent events and news with Gritstone ($GRTS) and Editas ($EDIT) - two very exciting biotechnology companies doing work in a variety of treatments like immunotherapy & gene editing.



Watch on YouTube

Initial clinical data for 101 is planned later this month. The data will include patient safety assessments and a preliminary analysis of secondary endpoints relating to signals of gene editing and clinical benefit. The presentation will cover cumulative data from patients in the adult low-dose and mid-dose cohorts. As required by the trial protocol, all patients are monitored every three months for the first year and at various timepoints for another two years.

Editas ($EDIT) has a Big Event Coming Up! | Gene Editing Biotech

Editas ($EDIT) has got a big event on September 27th, and I decided to ride the wave and share my thoughts as to why. Events are king in biotechs. Tell us what you think about our new series! Is this the kind if info you would be looking for? What other DD should we add?

Watch the Video on Youtube

Thank you for the explanation and your time. Have a good weekend.

Cancer is highly complex, so multiplexed gene editing will be needed to generate better cell therapies. One of the benefits of CRISPR is the ability to knockout multiple genes with (very) high efficacy, but that has not been the case for knock-ins. Now with this approach, it seems to have been overcome. If you take HLA-E, the efficacy (for knock-in) was over 88%, and this should 'shield' the majority of cells from being rejected by the patient they would be given to. ADAP will use the same approach for its iPSC-derived TCR-T cell therapies https://www.nature.com/articles/nbt.3860

Wow that sounds so?? What the heck does any of that mean?

''New preclinical data demonstrated that SLEEK results in the knock-in of multiple clinically relevant transgenes through a proprietary process that selects for cells containing the knock-in cargo. In addition, high percentage knock-in efficiencies were enabled by Editas Medicine’s proprietary engineered AsCas12a nuclease. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including iPSCs, T cells, and NK cells. Additionally, SLEEK may be used to fine-tune the expression levels of transgene cargos, an important attribute of next-generation cell therapy medicines.'' https://www.globenewswire.com/news-release/2021/08/20/2284285/0/en/Editas-Medicine-Presents-Data-on-New-SLEEK-Gene-Editing-Technology-at-Cold-Spring-Harbor-Laboratory-s-Genome-Engineering-CRISPR-Frontiers-Meeting.html

Slides https://www.editasmedicine.com/wp-content/uploads/2021/08/2021_CSHL-CRISPR-Frontiers-SLEEK-Zuris_FINAL.pdf

New preclinical data https://www.nature.com/articles/s41467-021-24017-8

Not sure what happened in the last hour, but the genomic stocks have all exploded upwards! EDIT is up 21% today. CRSP AND NTLA are making similar moves. I don’t know what triggered it, but good news for sure.

For sure! I've been in NTLA, EDIT and ARCT for a while just waiting for the CRSPR-Cas9 technology to be shown to be effective. We're just about there. If they don't explode in 2021, we should see it in 2022 the latest with all of the potentially great results from this technology!

Here we go! Gene editing stocks will be hot again. EDIT, CRSP, NTLA are going to be flying high. And this is technology that will be the future of medicine. Buy and hold. Buy calls when the price is right.

Ouch...LoL

EDIT has had a huge sell-off but should soon hit bottom. I can see a huge rebound starting within the next few days.

News out today ubx