[jondoeuk]

Upcoming https://www.globenewswire.com/news-release/2023/09/27/2750466/0/en/CRISPR-Therapeutics-to-Present-at-the-Society-for-Immunotherapy-of-Cancer-SITC-38th-Annual-Meeting.html

[jondoeuk]

$VRTX $CRSP FDA adcomm for exa-cel (sickle cell disease) set for Oct. 31.

Happy Halloween.

— Adam Feuerstein (@adamfeuerstein) September 6, 2023

[jondoeuk]

A new assessment released by the Institute for Clinical and Economic Review found that VRTX/CRSP's exa-cel and BLUE's lovo-cel would be cost-effective if priced between $1.35M and $2.05M https://icer.org/wp-content/uploads/2023/08/SCD_-RAAG_AUG-2023.pdf

Exa-cel has an FDA action date of Dec. 8, while lovo-cel's in Dec. 20. Regarding evidence of efficacy, ICER gave exa-cel a C++ rating, saying it would result in incremental net benefit, or result in substantial net benefit when evaluated against standard of care. For lovo-cell, the grade was B+, noting the treatment would provide at least an incremental benefit.

An independent appraisal committee overwhelmingly voted (13-1 for each therapy) that current evidence is adequate that both therapies provide a net benefit compared to the standard of care. ICER is also recommending that manufacturers and payers work together on alternative payment models for gene therapies. This is to address both the significant short-term budget impact of the treatments and the uncertainty surrounding their longer-term safety and benefits.

[jondoeuk]

2Q results https://uk.finance.yahoo.com/news/crispr-therapeutics-provides-business-reports-200100760.html

[jondoeuk]

No, but some anticipate a CHMP this year. As for the MHRA, I haven't heard anything.

[Monksdream]

CRSP bounced off the 200 day SMA

[Locomotive681]

Any idea on approval target date for the EU and UK?

[OldAIMGuy]

I added an extra 12% more shares to my CRSP inventory this AM at $55.09. That's a nice discount from where I last sold shares ($71.33).
The price/share is up about 7% from where I started this holding but through trading around the core I've managed to push up the holding's profits to 21%, including the cash reserve.


Best wishes,
OAG

[jondoeuk]

Data from EHA https://uk.finance.yahoo.com/news/positive-results-pivotal-trials-exa-070100851.html

Slides http://ir.crisprtx.com/static-files/d725fc70-6a5a-4ce2-a9b7-769b08955728

[jondoeuk]

FDA accepts BLAs for exagamglogene autotemcel for SCD and TDT. Grants Priority Review for SCD and Standard Review for TDT. The PDUFA date is 08/12/23 for SCD and 30/03/24 for TDT.

[janetcanada]

CRSP - Looks Promising.....

***Guide to the First Ever FDA CRISPR Approval Expected this Year by CRISPR & Vertex (XDNA ETF Companies)

APR 21, 2023 2:25PM EDT
Introduction

While being a newer technology, there are several publicly tradeable companies utilizing the power of CRISPR gene editing, base editing, and prime editing in the Kelly CRISPR & Gene Editing Technology ETF (Nasdaq: XDNA). Two of the XDNA companies are about to make history with potentially the first ever commercially available CRISPR technology.

***First FDA Approved Treatment - Major Milestone for the CRISPR Revolution: XDNA companies, CRISPR Therapeutics, 12.18%, and Vertex Pharmaceuticals, 1.56%, are anticipated to get the first ever U.S.-marketed treatment based on CRISPR this year for their Exa-cel therapeutic (data as of April 18, 2023).

CONTRIBUTOR
Kelly Intelligence
PUBLISHED

[Golden Cross]

Great future ahead for the technology

[janetcanada]

CRSP - Based on 17 analysts giving stock ratings to Crispr Therapeutics AG in the past 3 months

CRSP Stock 12 Months Forecast


?(17.05% Upside)
Based on 17 Wall Street analysts offering 12 month price targets for Crispr Therapeutics AG in the last 3 months. The average price target is $75.65 with a high forecast of $143.00 and a low forecast of $42.00. The average price target represents a 17.05% change from the last price of $64.63.

https://www.tipranks.com/

[OldAIMGuy]

Today's strong uptick triggered another GTC Limit Sell order for me. This one puts the holding nicely profitable with trades and price appreciation. With cash finally earning at least something, it helps total return on a holding that doesn't pay a dividend.



Best wishes,
OAG

[janetcanada]

CRSP - https://www.barchart.com/stocks/quotes/CRSP/overview

[janetcanada]

CRSP - CRISPR Therapeutics Revenues and Earnings Beat Expectations

***Earnings per share (EPS) also surpassed analyst estimates by 59%. Looking ahead, revenue is forecast to grow 58% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in the US.10 hours ago

CRISPR Therapeutics First Quarter 2023 Earnings

Yahoo
https://ca.finance.yahoo.com › news › crispr-therapeutics...
About featured

[JJ8]

Finally some positive action. Hopefully this is just the beginning of a price up move. The chart is looking good. GLTA

[OldAIMGuy]

Hi Bomber, Re: CRSP big move...................

Here's my response:


I let 10% of the position go yesterday which helps rebuild the cash cushion I carry for CRSP. That carried a tidy 44% LIFO gain with it and still leaves me with more shares than I started with a year ago. There's more cash on hand now, too.

Overall, the holding including the cash reserve is up 35% since the start by trading around the core investment. The price/share is up 25% since that start.

Best wishes,
OAG Tom

[rosemountbomber]

Big day today. Surprised no commentary here today.

[jondoeuk]

One of the benefits of using iPSC-derived is that after single cell cloning any DNA mutations/translocations are detectable in a clonal population, so they can be removed before a master cell bank is created.

[NY1972]

How much cancer risk is acceptable?

[janetcanada]

CRISPR Therapeutics to Participate Upcoming Investor Conferences

GlobeNewswire Inc.

CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team are scheduled to participate in fireside chats at the following investor conferences in May.
Bank of America Securities 2023 Health Care Conference
Date: Tuesday, May 9, 2023
Time: 10:40 a.m. PT

JMP Securities Life Sciences Conference
Date: Monday, May 15, 2023
Time: 11:30 a.m. ET

A live webcast of the fireside chats will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.gcs-web.com/events. A replay of the webcasts will be archived on the Company's website for 14 days following the presentation.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.

Investor Contact:
Susan Kim
[phone]
[e-mail]

Media Contact:
Rachel Eides
[phone]
[e-mail]

[jondoeuk]

CRSP's first major effort in this area (regenerative medicine), is in diabetes, and with ViaCyte, which was acquired by VRTX, are advancing a series of programs as part of a strategic collaboration for the discovery, development and commercialisation of gene-edited stem cell therapies for the treatment of it, starting in T1D. If that goes well, could move to insulin-dependent T2D.

They have filed extensive IP, are building a core set of capabilities, such as iPSC differentiation, along with manufacturing, which could allow the company to ultimately move to other organ systems, including the kidney and liver.

They began with VCTX210 and moved to VCTX211 (partnered 50:50 with VRTX). Starting with the former, used a smaller number of cells. The goal wasn't to achieve insulin independence or a significant reduction in insulin, but could they put the islet cells in a device, implant that under the skin, and do so safely in a handful of patients. They have enough confidence that allowed them to move to 211. It has been optimised in a number of ways, including the clone and cell dose, to actually produce enough insulin to potentially render these patients insulin independent. Also, has two additional edits, not obvious, A20 and MANF, genes that came out of large empirical screens to improve cell fitness.

Ultimately, they want a one an done procedure. But even if they get to a profile where a person has to have the device recharged every 2-3+ years, that should an acceptable profile from a market competitive standpoint.

[janetcanada]

CRSP - Looking Forward with Expectation to the FDA Approval!.......

Wishing a Good Weekend

Best of Luck
janet

[OldAIMGuy]

Thanks JC,
That would put CRSP at about a 10x gain from recent prices. That would be acceptable.

I've only had CRSP in my 'sandbox' portfolio for a year now. In that time I've trimmed the position 7 times and added to it 5 times. (small incremental trades around the core position)



The holding is currently around break-even but share count is now around 17% more than when I started a year ago. If the Roller Coaster continues to go up and down, I plan on sticking with the Ride toward that 2030 goal.

Best wishes,
OAG Tom

[janetcanada]

CRSP- CRISPR Therapeutics AG (NASDAQ:CRSP)

***Long-Term CRISPR Therapeutics - ***What is the CRISPR stock price forecast for 2030?

***Year Prediction Change
2028 $ 187.00 281.87%
2029 $ 244.47 399.23%
2030 $ 319.61 552.66%

***https://nz.finance.yahoo.com/

[jondoeuk]

Upcoming at ASGCT https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=13617

[jondoeuk]

EBMT oral (CTX110) presentation http://ir.crisprtx.com/static-files/2fddf877-a046-47fa-8428-614940a1cf7d

[janetcanada]

Vertex and CRISPR Therapeutics Announce Licensing Agreement to Accelerate Development of Vertex’s Hypoimmune Cell Therapies for the Treatment of Type 1 Diabetes
-Vertex to receive non-exclusive rights to CRISPR Therapeutics’ CRISPR/Cas9 to accelerate development of potentially curative cell therapies for T1D-

-CRISPR Therapeutics to receive $100M upfront payment plus milestone and royalty payments on potential future gene-edited hypoimmune T1D products-

BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Mar. 27, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20230324005339/en/

“We have multiple programs in our T1D portfolio including VX-880 and VX-264, which are in the clinic, as well as our hypoimmune program, in preclinical development,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “Having successfully demonstrated clinical proof of concept in T1D in our VX-880 program, we are excited to deepen our relationship with CRISPR Therapeutics with this agreement, which will allow us to further accelerate our goal of generating fully differentiated, insulin-producing hypoimmune islet cells for T1D.”

“We are pleased to expand our long and successful relationship with Vertex with this collaboration which fully leverages our gene editing platform to develop hypoimmune cell therapies for T1D,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “In parallel, we continue to expand our capabilities in regenerative medicine and advance our existing allogeneic gene-edited cell therapy programs.”

Under this agreement, Vertex will pay CRISPR Therapeutics $100 million up-front for non-exclusive rights to CRISPR Therapeutics’ technology for the development of hypoimmune gene-edited cell therapies for T1D. CRISPR Therapeutics will be eligible for up to an additional $230 million in research and development milestones and receive royalties on any future products resulting from this agreement.

CRISPR and ViaCyte, Inc., which was acquired by Vertex in 2022, will continue to collaborate on their existing gene-edited allogeneic stem cell therapies, using ViaCyte cells, for the treatment of diabetes under the terms of their collaboration. A Phase 1/2 study of VCTX211, an allogeneic, gene-edited, stem cell-derived product candidate for T1D, which originated under the CRISPR Therapeutics and ViaCyte collaboration, has been initiated and is ongoing. CRISPR Therapeutics will not obtain any interest in Vertex’s pre-existing pipeline of T1D products, including VX-880 and VX-264.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, and alpha-1 antitrypsin deficiency.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s Best Workplaces in Biotechnology and Pharmaceuticals and Best Workplaces for Women. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

(VRTX-GEN)

Vertex Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Bastiano Sanna, Ph.D., and Samarth Kulkarni, Ph.D., in this press release, statements about the terms of and expectations for Vertex’s collaboration with CRISPR, potential benefits and results that may be achieved through the collaboration, including acceleration of the development of Vertex’s hypoimmune cell therapies for T1D, statements regarding the future activities of the parties pursuant to the collaboration, statements regarding upfront and milestone payments, and potential royalties on future products, and statements regarding the collaboration between CRISPR and ViaCyte and that CRISPR will not obtain any interest in Vertex’s pre-existing pipeline of T1D products. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of Vertex’s collaboration with CRISPR may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading “Risk Factors” in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through Vertex’s website at www.vrtx.com and on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.

CRISPR THERAPEUTICS® word mark and design logo and VCTX211™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

(CRSP-GEN)

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including without limitation, statements made by Samarth Kulkarni, Ph.D. and Bastiano Sanna, Ph.D. in this press release, as well as statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) the future activities of the parties pursuant to the ViaCyte collaboration and the expected benefits of such collaboration, including expectations regarding VCTX211; (ii) the safety, efficacy and progress of its clinical programs; (iii) upfront and milestone payments, and potential royalties on future products, under the non-exclusive license; and (vii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: that it may not realize the potential benefits of its collaborations on the anticipated timeline, or at all; the potential that clinical trial results may not be favorable; that one or more of product candidate programs will not proceed as planned for technical, scientific or commercial reasons; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics’ most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.


View source version on businesswire.com: https://www.businesswire.com/news/home/20230324005339/en/

Vertex Pharmaceuticals Incorporated

Investors:
[e-mail]
Susie Lisa, [phone]
or
Manisha Pai, [phone]
or
Miroslava Minkova, [phone]

Media:
[e-mail]
or
U.S.: [phone]
or
Heather Nichols: [phone]
or
International: [phone]

CRISPR Therapeutics

Investors:
Susan Kim, [phone]
[e-mail]

Media:
Rachel Eides, [phone]
[e-mail]

Source: Vertex Pharmaceuticals Incorporated

[janetcanada]

CRSP - Interesting Video from Dylan Jovine

DYLAN JOVINE CLAIMS THE NEXT BIG BIOTECH TAKEOVER IN 2020 WILL BE A” CUT AND PASTE” COMPANY

"This report tells you everything you need to know about this incredible company --- and why the world’s leading scientist believe its CRISPR technology is the best, giving you the chance to for a 46,751% return"


<< It could help you achieve the kind of carefree retirement most people only dream of. But what if this turns out to be your biggest opportunity? How long will you stay in the dark>>? Continue reading...

[jondoeuk]

Based on the data for CTX110 (a first-gen CD19-targeted CAR-T), and discussions with regulatory agencies, the company initiated a PhII single-arm potentially registrational trial, which incorporates consolidation dosing.

Also, could have feedback from regulatory agencies for CTX130 (another first-gen CAR-T, but CD70-targeted) mid-year. I know they are hoping to move it into another registrational trial in certain R/R T-cell lymphomas.

[NY1972]

Another bio with endless research in crowded field, can't beat AUTO, can't salvage AUTO relapsed. Just lots of hype from crispr to keep printing shares.

[jondoeuk]

Slide deck http://ir.crisprtx.com/static-files/ce2efeb9-3319-4504-8430-13058b305455

[jondoeuk]

The therapies for SCD could be cost effective even with a $2M price tag, the ICER says in a draft report analysing two https://endpts.com/at-2m-per-shot-icer-says-two-new-sickle-cell-gene-therapies-could-be-cost-effective/

[Windbag1014]

Investment analysts at Cantor Fitzgerald started coverage on shares of CRISPR Therapeutics (NASDAQ:CRSP - Get Rating) in a research note issued to investors on Thursday, The Fly reports. The brokerage set an "overweight" rating and a $72.00 price target on the stock. Cantor Fitzgerald's price target suggests a potential upside of 65.63% from the company's current price.

https://www.marketbeat.com/instant-alerts/nasdaq-crsp-a-buy-or-sell-right-now-2023-04-13/

[jondoeuk]

Both are now recruiting. For the CTX131 trial (n=250) unlike CTX130, which was tested in just patients with R/R ccRCC, 131 is open to patients with cervical, esophageal, pancreatic, or malignant pleural mesothelioma.

[jondoeuk]

An historical milestone https://finance.yahoo.com/news/vertex-crispr-therapeutics-complete-submission-120000960.html

The companies are requesting Priority Review.

[jondoeuk]

VRTX has signed a licensing deal to accelerate development of its hypoimmune cell therapies for the treatment of T1D https://www.fiercebiotech.com/biotech/vertex-goes-back-crispr-well-paying-100m-upfront-exa-cel-partner-help-diabetes-work

[janetcanada]

CRSP - From: Simply Wall St™

***Revenue is forecast to grow 49.88% per year

*Pass
Are revenue and earnings forecast to grow?

Earnings are forecast to grow by an average of 52.4% per year for the next 3 years
Section 2.1

*Pass
Are they in a good financial position?

They have sufficient cash for at least 1 year of operations
Section 4.4

Pass
Is their dividend sustainable?

They do not pay a dividend
Section 5.0

*Pass
Is their share price liquid and stable?

Share price has been stable over the past 3 months

Pass
Do they have sufficient financial data available?

They have sufficient analyst coverage
Section 2.0

Pass
Are there any concerning recent events?

No concerning events detected

Pass
Do they have a meaningful market capitalization?

Market cap is meaningful ($4B)
Section 8.0

*Pass
Do they have negative shareholders equity?

CRSP does not have negative shareholders

***CRISPR Therapeutics NasdaqGM:CRSP Stock Report

***Simply Wall St
https://simplywall.st › ... › Pharmaceuticals & Biotech
====================================================

***Founded Employees CEO Website
2013 458 Sam Kulkarni https://www.crisprtx.com
CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. Its CRISPR/Cas9 is a gene editing technology that allows for precise directed changes to genomic DNA. The company has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases.

The company’s lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta-thalassemia or severe sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It also develops CTX110, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19; CTX120, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma; and CTX130, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting Cluster of Differentiation 70 to treat various solid tumors and hematologic malignancies. In addition, the company develops VCTX210, a gene-edited immune-evasive stem cell-derived product candidate for the treatment of treatment of type 1 diabetes; and VCTX211, an investigational, allogeneic, gene-edited, stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics.

CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.

[jondoeuk]

This will be an oral presentation (of preclinical data) at AACR next month.

Using AI and machine learning, they did a very simple set of experiments in a robust fashion, which is to take thousands of different edits without any bias and to do them pairwise to see what was going to increase the potency of the CAR-T cells. One of those edits is TGFBRII knockout and the other one is a Regnase-1 knockout. Not only did the latter come out of a large empirical screen in one of the partnerships the company had, but other groups have shown each can improve antitumour activity https://insight.jci.org/articles/view/133977 https://www.nature.com/articles/s41586-019-1821-z

So that combination of edits in preclinical testing, is at least tenfold better, if not more. Also, in their manufacturing process, the expansion of these cells is a lot greater, and the CEO has said they have seen a greater proportion of the central memory genotype phenotype https://ashpublications.org/blood/article/127/24/2980/35453/Phase-1-studies-of-central-memory-derived-CD19-CAR

When they start dosing patients, will know quickly what the PK/PD profile looks like for CTX112 and CTX131. If the expansion at DL1, let's say, is tenfold higher for these CAR-T's, the therapy should be more potent and work better.

Using CTX131 in R/R ccRCC, the first thing will be those expansion kinetics and the second will be responses. Assuming those responses are at low doses than with CTX130 it would inform them as to how they will think about investments, because if they start seeing those signs, will be going to go pedal to the metal on other CAR constructs and targets, with the off-the-shelf platform, especially now they have their own manufacturing.