For the development of CRISPRCas9 as a method for genome editing along with the collaborator, Doctor. Jennifer Doudna. Over the past 5 years, we have made tremendous progress in building the leading CRISPR company with 4 clinical programs, over 300 employees and over $1,000,000,000 in cash balance.
Good morning, and welcome, ladies and gentlemen, to the CRISPR Therapeutics Clinical Update Conference Call. At this time, I would like to inform you that this conference call is being recorded and that all participants are in a listen only mode.
Allogeneic CAR T programs into the clinic in just 4 years, the first of which we will talk more about today. We could not have accomplished all of this without the Ryerson's
pleased to report
that Good morning and thank you for joining us as we report results from the CARBON trial, an ongoing Phase 1 study of CTX-one hundred and ten, a healthy donor derived gene edited allogeneic CAR T therapy for the treatment of CD19 positive B cell malignancies. We recommend that you access the webcast slides on our website as you listen to this call. This conference call is being recorded and a replay will be available on our website. During today's call, we will be making certain forward looking statements. These statements may include statements regarding, among other things, the efficacy and safety of our product candidates, our research and development plans, regulatory plans and our plans to present or report additional data.
These forward looking statements are based on current information, assumptions and expectations that are subject to change and involve risks and uncertainties that may cause the actual results to differ materially from those contained in the forward looking statements. These and other risks are described in our periodic filings made with the SEC, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward looking statements, which are only made as of today's date. The company disclaims any obligation to update such statements. Joining me this morning are Sam Kulkarni, our Chief Executive Officer Evelina Maraba, our Vice President of Clinical Development Doctor.
Joe McGurk, Professor of Medicine and Division Director of Hematologic Malignancies and Cellular Therapeutics at the University of Kansas Medical Center and one of the investigators on the study and Tony Ho, our Head of Research and Development. With that, I'm pleased to turn the call over to Sam Kulkarni, who will make some introductory remarks.
Thank you, Susie. Good morning and thank you all for joining us today. Earlier this morning, we reported the first clinical results from our Phase 1 CARBON trial of CTX-one hundred and ten for the treatment of B cell malignancies. We are proud to share this data just a few days after our scientific founder, Doctor. Imman's company to test the beta-two ms knockout strategy as a means of allowing allogeneic persistence without the need for more toxic preconditioning regimen, Jennifer Doudna.
Over the past 5 years, we have made tremendous progress in building the leading CRISPR company with 4 clinical programs, over 300 employees and over $1,000,000,000 in cash balance. In 2019, we were the first to demonstrate the power of CRISPR gene editing in patients with rare diseases in the
CARBON trial, which is currently open to enrollment globally at 7 participating institutions on 3 continents. Carbon is enrolling adult patients with the CLL.
Building beyond CTX-one, we have advanced 3 gene edited allogeneic CAR T programs into the clinic in just 4 years, the first
of which we will talk about.
Patients received 3 days of lymphodepleting chemotherapy consisting of fludarabine at 30 milligrams per meter squared and cyclophosphamide at 500 milligrams per meter squared, followed by TTX-one hundred and ten infusion. Some patients have completed screening in as few as 3 days and all patients began LD chemotherapy within a median of 2 days of enrollment. This timeline is possible because there's no need for apheresis or bridging chemotherapy. Oncology
based on the belief that multiplex gene editing, which CRISPR enables to a greater extent than earlier technologies, will be essential to capturing the full a allogeneic CAR T in which the T cells from a healthy donor are used as the starting material instead of a patient's own cells could overcome many of the changes that
we have
been enrolled in this study and all have received CTX-one hundred and ten. Dose escalation began at 30,000,000 CAR positive T cells or dose level 1 and escalated in approximately 2 or 3 fold increments to the highest dose of 600,000,000 CAR positive T cells, which is dose level 4. At each
we believe that over time, allogeneic therapies represent a whole new class of medicines that can become the first line of defense in the fight against cancer. Creating a safe and effective allogeneic CAR T therapy requires the editing of multiple genes within the T cell simultaneously.
11 patients have had at least 28 days of post treatment follow-up.
Continuous added to the genome of healthy donor T cells that are designed to avoid graft versus host disease, target the CAR T cells of the tumor and evade a patient's immune system. To our knowledge, we are the 1st company to test the beta-two ms knockout strategy as a means of allowing allogeneic persistence without the need for more toxic preconditioning regimen. I will now turn the call over to Doctor. Evelina Morava, our Vice President of Clinical Development and Doctor. Joe McGurk, Professor of Medicine at the Kansas University Medical Center, who will go through a presentation of initial data from the CARBON trial.
Thank you, Sam. Dose escalation of CPX-one hundred and ten is being performed in the CARBON trial, which is currently open to enrollment globally at 7 participating institutions on 3 continents. CARBON is enrolling adult patients with relapsed or refractory large B cell non Hodgkin lymphoma who have failed at least two lines of therapy. Early evidence of dose dependent responses was seen with CTX-one hundred and ten. Efficacy assessment was performed by centralized independent at 30 milligrams per meter squared and cyclophosphamide at 500 milligrams per meter squared, followed by CTX-one hundred and ten infusion.
Some patients have completed screening in as few as 3 days, and all patients began LD chemotherapy within a median of 2 days of enrollment. This timeline is possible because there's no need for apheresis or bridging chemotherapy on this trial And CTX-one hundred and ten is available at the site before a patient is enrolled. The primary objective of CARBON is to evaluate the safety and efficacy of escalating doses of CTX-one hundred and ten. After a dose of CTX-one hundred and ten is determined, the trial will proceed seamlessly into a potentially registrational single arm efficacy expansion cohort. As of September 28, 2020, 12 patients have been enrolled in the study and all have received CTX-one hundred and ten.
Those escalation began at 30,000,000 CAR positive T cells or dose level 1 and escalated in approximately 2 or 3 fold increments to the highest dose of 600,000,000 CARP. We have a percentage change in tumor size for patients treated with CTX-one hundred and ten across the 4 dose levels. The color of the bars reflects the dose level at WICUs. No DLTs were observed in dose levels 1, 2 or 3. After the initial cohort of 3 patients were treated at dose level 3, the encouraging anti tumor activity led us to enroll additional into both dose level 3 and dose level 4 cohorts simultaneously.
11 patients per nodal disease, normalization of all nodal disease to 1.5 centimeter or smaller and a Dolbyl score of 2 or lower. Additionally, the study continues to enroll currently. This table summarizes the baseline characteristics for patients enrolled in CARBON across the 4 dose levels. These patient characteristics are consistent with those in other clinical trials evaluating autologous or allogeneic CD19 response and follow-up. Median follow-up time for the 11 patients was 1.8 months.
All patients treated at dose level 2 or 3 who had a complete having received at least two lines of therapy, including 4 patients with prior autologous transplant. The majority of patients had advanced stage lymphoma and were refractory to their last line of therapy before entering the study. The median time from last therapy to the start of LD chemotherapy for all 11 is planned to be redosed at dose level 3. The patient at dose level 4 that achieved a complete response at month 1 has subsequently died due to an adverse event after data cutoff and will be discussed in more detail later. This table summarizes the safety data for the 10 patients treated at dose level 3 and below that occurred following CTX-one hundred and ten infusion.
First, there have been no cases of GvHD despite the high HLA mismatch between CAR T or commercial CD19 directed CAR T products. Although dose level 1 was determined to be sub efficacious, complete responses were obtained at dose level emergent adverse events of special interest. 3 patients treated with CTX-one hundred and ten have experienced a total of 4 events of CRS. 2 of these events 2 of the 4 patients had a complete response. To date, all of the objective responses in the study have been complete responses and we're seeing that the 1st Grade 2 eye counts occurred in one patient and improved within 24 hours of steroid administration.
2 additional treatment emergent in 2 patients with transformed follicular lymphoma as well as in patients who are primary refractory and who had relapsed after autologous dental transplant for TROPENIA. In summary, these preliminary data suggest an acceptable safety profile at dose level 3 and below with no events of graft versus host disease or grade 3 or higher of CRS or ICANN. The one patient recently treated at dose level 4 with a 70 shows evidence of decreasing tumor size and that the deeper responses are associated with higher doses of CTX-one hundred and ten. The 4 patients with complete responses had complete resolution of external disease, normalization of all nodal disease to 1.5 centimeter or smaller and a Dolbyl score of 2 or lower. Additionally, one of the patients with a complete response achieved complete clearance of 30% when 2 was admitted for febrile neutropenia on day 26 and shortly after on day 28 began to have short term memory loss and confusion.
The symptoms eventually progressed to significant optimization that required intubation. The patient underwent several diagnostic procedures, including lumbar puncture and brain MRI. He was initially treated for ICANN with high dose steroids andakinra and intracycl chemotherapy without any improvement. He was at dose level 2 who achieved a complete response was found to have recurrence of disease on imaging approximately 5.75 months after CTX-one training showed that he was HHV-six IgG positive consistent with prior HHV-six infection. The diagnosis of HHV-six encephalons at month 1 has subsequently died due to an adverse event after data cutoff and will be discussed in more detail later.
This table summarizes status and at the request of HD despite the high HLO mismatch between CAR T donors and patients. 2nd, no infusion reactions to either lymphodepleting chemo therapy or CTX-one hundred and ten have occurred. Focusing on treatment emergent adverse events of special interest, 3 patients treated with CTX-one hundred and ten have experienced a total of 4 events of CRS. 2 of these events were Grade 1 CRS per AFDCT criteria and required no intervention. 2 events were Grade 2 CRS and resolved with the use of tocilizumab.
Grade 2 ICANS occurred in one patient and improved within 24 hours of therapy.
We filed
CTX-one hundred and ten in dose level 2 to 4. CTX-one hundred and ten cells were detected in all patients as measured by PCR. Following CTX-one hundred and ten infusion, there was a transient decline in CTX-one hundred and ten, which can be attributed to distribution of cells into compartments. Peak at dose level 3 and below with no events of graft versus host disease or grade 3 or higher of CRS or iCAM. The one patient recently treated at dose level 4 was a 73 year old man with transformed follicular lymphoma who relapsed following autologous stem cell transplant.
I see close of data and will provide further updates at a future scientific meeting. And with that, I would like to turn the call over to Doctor. McGurk, who will present and respond to tocilizumab. On day 25, the patient underwent his 1 month scan and was found to the incomplete response. After the 1 month scan, the clinical course became complicated.
The patient was admitted for febrile neutropenia on day 26 and shortly after on day 28 began to have short term memory loss and confusion. The symptoms eventually progressed to significant optimization that required intubation.
The patient underwent a significant treatment for the treatment of the
treatment of the treatment of the treatment of the
treatment of the treatment of the treatment of the treatment of the treatment of the
treatment of the treatment of the treatment of the treatment of the treatment of the treatment of the treatment of
the treatment of the He had an uncomplicated course after CTS-one hundred and ten infusion, did not have any CRS, eye cans or infection. The patient had a clear response to the CTS-one hundred and ten on physical exam with decreasing size
of the patient's blood collected at screening show that he was HHV-six IgG positive consistent with prior HHV-six infection. The diagnosis of HHV-six encephalitis was based on CSF examination that revealed a high HHV-six titer of approximately 650,000 copies per ml. Subsequently, he began a course of antiviral therapy, which continued for 13 days.
Per Lugano criteria, this would be considered stable disease. At that point, we decided to obtain a lymph node biopsy
at day 52 post CTX-one hundred and ten infusion. We believe the exact ideology of neurological deterioration is likely due to a combination of HHV-six reactivation in an immunocompromised patient that led to HHV-six encephalitis and potentially ICANN. In discussion with the we delivered the CD19 positive population
and only the CD19 negative remained and gave rise to subsequent persistence of disease. Although disappointing, this case speaks to the on target activity of CTX-one hundred and ten and could potentially be addressed by future iterations of CAR T products
with the CRISPR study.
This is in all patients as measured by PCR. Following CTX-one hundred and ten infusion, there was a transient decline in CTX-one hundred and ten, which can be attributed to
a 62 year old woman who was diagnosed with diffuse large T cell lymphoma. She had initially received 6 cycles of R CHOP achieving only a partial response. She then received rice salvage chemotherapy and similarly received only a partial response before receiving fusulfan cyclophosphamide conditioning followed by an autologous stem cell transplant. This year, she relapsed and was referred to our center for further management. She presented with an approximately 6 centers.
With that,
I would like to turn the call over to Doctor. McGurk, who will present case studies on 2 of his patients treated with PTX-one hundred and ten.
Thank you, Evelyn. She was enrolled on this trial and treated at dose level 3. She had an uncomplicated course without any CRS, eye cans or infection. I'm excited to have a number of patients who have been
in the
hospital for a long time.
She was a 62 year old male with transformed follicular lymphoma who previously received 2 lines of therapy including R CHOP and dose adjustment for a 3 month follow-up and she remains in complete remission with no toxicity.
Thank you, Doctor. McGirt.
We are very encouraged
by this the CTX-one hundred and ten infusion did not have any CRS, eye cans or infection. The patient had a clear response to the CTX-one hundred and ten on physical exam with decreasing size of cervical lymph nodes. In fact, I can see this patient's lymph nodes from across the exam room
and at day 8 following 4 patients. While we have seen early signs of durability, we will continue to monitor the ongoing complete responses. We see an acceptable safety profile at dose level 3 and below with no GVHD and low grades and rates of CRS and ICANN. Furthermore,
this would be considered stable disease.
Already becoming evident with 100% of enrolled patients receiving treatment without need for bridging chemotherapy or worry about manufacturing failures. Furthermore, we observed responses across multiple lots of CCX-one hundred and ten manufactured from different donors, suggesting that as anticipated this
suggests that the CAR T cells had cleared the CD19 positive population and only the CD19 negative remained and gave rise to
the next slide.
And it makes us excited to progress CTX-one hundred and ten as well as CTX-one hundred and twenty, CTX-one hundred and thirty and additional allogeneic CAR T programs forward as rapidly as possible. To that end, we are moving full thing ahead on CTX-one hundred and ten. We plan to proceed into an expansion cohort in the near term following Sebastian with an optimal dose. In addition, we have now included redosing as an option for patients who relapse or do not achieve a complete response. And we have already redosed 1 patient.
We believe that the ability to redose
before receiving the sulfam cyclophosphamide conditioning followed by an autologous stem cell transplant. This year, she relapsed and was referred to our center.
We continue to make rapid progress on CTX-one hundred and twenty and CTX-one hundred and thirty with dosing ongoing across trials in multiple implant
confirmed to be diffuse large B cell lymphoma on biopsy. She was enrolled on this trial and treated at dose level 3. She had an uncomplicated course without any CRS, eye cans or infection. I'm excited to report that in 1 month assessment, there was no evidence of lymphoma by either PET or CT. The subject is clinically very well.
I just saw her about a month ago for a 3 month follow-up and she remains in complete remission with no
toxicities. 4 years ago, we set out a concrete strategy. 1st, validate the allogeneic platform with proven targets, then expand from hematologic cancers into solid tumors, And finally, unlock the full potential of immuno oncology cell therapy through CRISPR multiplex tumor activity with complete responses achieved in 4 patients. While we have seen early signs of durability, we will continue to monitor the unachiev, our first step towards accomplishing this ambitious goal by validating our allogeneic platform in the clinic. We are looking forward to an ICANN.
Furthermore, no additional lymphodepleting agents beyond a standard flu site regimen were required to achieve these responses. Finally, the advantage of the allogeneic approach are already becoming evident with 100% of enrolled patients receiving treatment without need for bridging chemotherapy or worry about manufacturing failures. Furthermore, we observed responses across multiple lots of CTX-one hundred and ten manufactured from different donors, suggesting that as anticipated, this approach can scale to benefit as many patients as possible. In law, we believe this data validates our CRISPR edited allogeneic CAR T approach and makes us excited to progress CTX-one hundred and ten as well as CTX-one hundred and twenty, CTX-one hundred and thirty and additional allogeneic CAR T programs forward as rapidly as possible. To that end, we are moving
congrats on the updates and thanks for taking my questions. I just had a one two part question. So I'm wondering if you can In
addition, we have now included redosing as an option for patients who relapse or do not achieve a complete response. And we have already redosed 1 patient. We believe that the ability to re dose with CTX-one hundred and ten provides an additional advantage that will increase our ability to achieve durable complete responses in as many patients as possible. We continue to make rapid progress on CTX-one hundred and twenty and CTX-one hundred and thirty. With dosing ongoing across trials in multiple and myeloma, renal cell carcinoma and T and B cell lymphoma.
We expect
to And the NK cells as well as ICANS. We are collecting all sorts of information on these patients whether they're
TCX-one hundred and thirty which includes both novel targets and novel edit. And we plan to announce additional programs in 2021. When we initiated our allogeneic CAR T efforts 4 years ago, we set out a concrete strategy. 1st, validate the allogeneic platform with proven targets, then expand from hematologic cancers into solid tumors, and finally, unlock the full potential of immuno oncology cell therapy through CRISPR multiplex editing. Our IO pipeline reflects that strategy.
Today,
potentially increase the risk of ICANNDS. In addition, the inclusion exclusion criteria for our trials is very similar to what's been done with other
We are looking forward to continuing to innovate novel CAR T designs and investigating them in the clinic with the aim of bringing multiple transformative off the shelf CAR T therapies to patients. None of this would be possible without our investigators, site staff and above all, patients and their families who have had the courage to participate in this groundbreaking clinical review.
How many patients with follicular lymphoma versus the DLBCL, particularly in the dose level
We'll take our first question from Maury Raycroft of Jefferies.
Hi, good morning, everyone. Congrats on the updates and thanks for taking my questions. I just had a one two part question. So I'm wondering if you can talk about whether you're seeing NK cell proliferation or activity. And is this related to CRS or any other safety observations?
And for the grade 2 ICANS or other SAEs, can you provide more specifics or comments on potential influence from baseline characteristics, including CNS involvement or number of immune cells in CSF at baseline? And can you mitigate for these issues going forward?
Yes. Thank you, Maury. We're quite excited to present these data today and very excited about the complete responses we're seeing. To your two questions on the pharmacokinetics and the NK cells as well as ICANS, we are collecting all sorts of information on these patients, whether they're cell proliferation of our CAR Ts, what happens to the host immune system, and at the right medical meeting, we'll present all the data holistically. So you'll see more on that.
We're very excited though the fact that our CAR Ts persist for a very long time and can be detectable up to 180 days, which was one of the key hypotheses in making the beta-two ms edit. I think It
looks like the complete response from the dose level 2 patient was about out to 6 months or so.
Is that what you would also expect to
For 6 that we have noted thus far that potentially increase the risk of ICANN, in addition, the inclusion exclusion criteria for our trials is very similar to what's been done with other, with other autologous CAR T cell trials where ICAMS has been seen as well.
Our next question is from Gena Wang of Barclays. Your line is open.
Thank you for taking my question.
Such a long response at DL2, which is a suboptimal dose, gives us great confidence that when we see deeper responses at DL3.
I think I heard
it follicular lymphoma. I just wanted to confirm how many patients were follicular lymphoma versus the DLBCL, particularly in the dose level, stage 2 to 3? And then also regarding more the technical part, I wanted to ask you regarding the co stimulatory domain, was that CD28 or 41BB? And then lastly, very quickly on the IP, do you have IP on beta-2M knockout technology?
Yes. Thank you, Gina. And I'll start with your last question first, which is we've filed extensively in terms of IP on our CAR T constructs, whether it's the beta-two ms edit or other aspects. CRISPR gene editing just makes better quality T cells and CAR Ts. They're healthier and more robust in terms of cytotoxicity.
So that's something that the top line answer and then I'll ask Evelina to provide more details here. I think the N is small, but I think it's very encouraging. I think of the we again protect the overall construct with IP with many different filings that you'll see over time. I think DLBCL or TFL or whether they're primary refractory or relapse gives us great confidence. I think this is a very important update for the chart.
And I just want to emphasize that transformed follicular aspect, we're looking at very serious follicular patients, not indolent follicular patients here. And we see complete responses in both DLBCL and transformed follicular.
We'll take our next question from Geoff Meacham of Bank of America.
Hi, guys. This is Greg Harrison on for Geoff. Thanks for taking the question. So it looks like the complete response from the dose level 2 patient was about out to 6 months or so.
Is that what you would also expect to see in
the ongoing CRs in both Level 3 or is there any reason you can believe?
As Sam mentioned, our hope and we're incredibly encouraged by what we're seeing right now that redosing may not be necessary, but it's an option on our trial.
On durable responses, let me just remind you and contextualize the fact that durability ultimately is a function of both how deep the responses are
and the 12 patient dose was on dose level 3.
Gives us great confidence that when we see deeper responses at DL3 and above, we eventually should translate into durability in the clinic.
Are those learnings from this study that you think can apply to the other CAR Ts? And how do you see this as building the foundation to start to do multiplex with respect to additional targets? Thank you so much.
Yes. Thank you, Ted. And I'll start and then I'll Thank you for
taking my question. Just given the small ends in each of the dose cohorts, can you just discuss your work on the forward here to better determine dose?
For folks, I think as a company, we only were have been around for 5 years, it's a brick and mortars company. And within that time, we came up with the CTX-one program, disclosed that data last year, which can be transformative. And then in parallel, I've developed a host of 3 different CAR Ts, which are all in clinical trials right now and enrolling. Thank you, Salveen. I'll start with some with a top line answer and then I'll ask Abelina to provide more details here.
I think the end is small, but I think it's very encouraging. I think of the 4 patients that we've dosed at dose level 3 and the patient at dose level 4, I think seeing the complete response was in these patients, whether they're DLBCL or TFL or whether they're primary refractory or relapse, gives us great confidence. I think this is a very important update for the trial as we look to expand the number of sites, as we look to bring more patients in. And this will be transformative and have an important place in the lines of treatment for these patients that have limited options. I think beyond that, I think the thesis with the hope that that can become the registrational trial, assuming the data continue to hold.
I think we haven't said exactly what that expansion dose is going to be and we will continue to update the street as we go along in the trial. But at this point, I think we are full steam ahead on this trial once we complete the dose response and we want to get into expansion. I think to the second question around the redosing of that patient, I will turn it over to Evelina.
Thanks, Sam. So as Sam mentioned, our hope and we're incredibly encouraged by what we're seeing right now that redosing may not be necessary, but it's an option on our trial and we provide this option to patients that have either relapsed or patients that have had some kind of a response, but not full complete remission. And the 12 patient dose was on dose level 3.
Our next question is from Ted Tenthoff of Piper Sandler.
Great. Thank you very much. Positive update. Are there learnings from this study that you think can apply to the other CORTES and Raju Prasad of William Blair.
Thanks for taking the question. Just a couple of questions on the SOL product.
Yes. Thank you, Ted. And I'll start and then I'll ask Tony to make some comments as well. But I think this is very exciting for
with regards to peak or durability and your responses. And then anything you can say on percent of the B2M knockout or knockdown?
And within that time, we came up with the CTX-one program, disclosed that data last year, which can be transformative. And then in parallel have developed a host of 3 different CAR Ts, which are all in clinical trials right now and enrolling rapidly. I think we're moving along on CTX-one hundred and twenty, which is targeted towards BCMA regardless of the healthy donor lots that we're using more than one. And obviously, we take a lot of robust characterization to characterize this at with CTX-one hundred and ten, does give us great confidence in what we may see with CTX-one hundred and twenty and CTX-one hundred and thirty. We will have data updates for those 2 programs next year, as well as additional data on CTX-one hundred and ten.
I think that, one, gives us confidence in negative. I think we will disclose that's very important data. And as you can imagine, it's highly competitive data. I think the beta-two EMEDITH gives us a distinct advantage over other players in the allogeneic field and we're filing IP on various aspects of it as we go along. We will disclose the data of how the cells behave with or without beta2M edits.
But it's very encouraging, as Tony was saying, that we see these cells be continuing work and how we see this over the next 5, 10 years.
Sure. I think as you have seen, our initial data give us
a lot of confidence providing holistic data at the medical meeting where we show the comparison, the pharmacokinetics, etcetera.
Both blood and the bone marrow out to day 180, really sort of validated our allogeneic immune based platform. So we're very excited to sort of build on the chassis to add additional edits and also additional target. These could be added that resist a tumor microenvironment, exhaustion to resistance and select targeting and also we can build things that elicit immune endogenous immune response. So I think this is just a start. We're looking forward to continue to build our platform.
For the cell type versus responders or non responders, We haven't disclosed that information at this point, but I think we did show a chart that showed the cell expansion chart and you do see a curious expansion in the 1st 7 to 10 days, you see a lot of the expansion happening. And I think a lot of
And any signs you're seeing kind of engraftment with regards to peak or durability in your responses? And then anything you can say on percent of the B2M knockout or knockdown would be helpful? Thanks.
Thank you, Raju.
I think what we did disclose Certainly, we're still learning from these data. Most importantly, we saw CTX-one hundred and ten were detecting multiple time points all the way
to day 180. We are seeing effect in patients and responses regardless of the healthy donor lots that we're using more than 1. And obviously, we take a lot of robust characterization to select healthy donor lots that we want for this manufacturing. But we have used multiple healthy donor lots to make these drug products and we're seeing responses across multiple lots. So that's very encouraging.
I mean chemotherapy and able create a window for CTX-one hundred and ten expansion and as Stan was saying, we will continue
to do that. I think the beta-two imedit gives us a distinct advantage over other players in the allogeneic field and we're filing IP on.
Our next question is from Arlinda Lee, Canaccord Genuity.
Hi, guys. Congratulations on
But it's very encouraging, as Tony was saying, that we see these sales persist for a very long time. I think finally to your question on Have
you seen anything in your 120 or 130 with regards to that or have you changed your protocols with regards to that? And then maybe for Doctor. McGirt, how do you see the use of 110 to get a bridge to another treatment or are you looking for greater durability in the future?
Thank you very much, and congrats on the data. I was wondering if
you could provide any additional color on the expansion that you're seeing. And I guess, are you seeing better expansion in patients who
clinical perspective, in a program that has done a lot of cell therapy now in clinical trials and in the commercial space, we see this as the next generation of therapies for our patients with specific advantages to these constructs and that it's we think importantly a more fit population of T cells that have not failed to control tumor growth in the first place because they are not from the patient.
At 10 days, you see a lot of the expansion happening. And I think a lot of the activity of the CAR T is happening very early on. And that is an interesting profile for these allogeneic therapies. But let me turn over to Tony and see if he has any additional comments to make. But the holistic data presentation will come.
So we are very much encouraged with the signals that we are seeing in terms of both efficacy and each early dose early dose detecting multiple time points all the way to day 180
in our patients. And following CTX-one hundred and ten infusion, there was a transient decline of CTX-one hundred and ten, which can be attributed to the distribution of these cell to different compartments, including the tumor. Then we saw peak expansions you have seen on the curve occurs for approximately 1 to 2 weeks after administration. We're encouraged to use this as the
next generation, this is the future for our patients. That's why we're participating in this important study.
Thank you, Doctor. McGurk. And Arlinda, to the second part of your question around the eye cancer, I think we Evelina disclosed the course of the clinical complications and the course of treatment for that patient.
Understood. Congrats again. Thank you.
Our next question is from Arlinda Lee, Canaccord Genuity.
Hi, guys. Congratulations on the data. I have a couple of questions. Maybe I could talk
a little bit further about the headcount. And then maybe for Doctor. McGirt, how do you see the use of Y penetrated bridge to another treatment? Or are you looking for greater durability?
Thank you, Arlinda. Let me start with your second part of your question first and turn it over to Doctor. McGurk on how he sees CTX.
On the safety side, were the different doses associated with different levels of It's just subsequent therapy.
From a clinical perspective, in a program that has done a lot of cell therapy.
And dose level 2 is not the patient that was re dosed. I previously mentioned to one of your colleagues that you previously mentioned that the patient that was re dosed with them.
And Ed, it's we think importantly a more fit population of T cells that have not failed to control tumor growth in the first place because they're not
I think dose level 4 was an unfortunate case that occurred and had very complicated neurological course that was both
a result of lymphocytes. Very specific, we think it's a more physiologically active less likely to become exhausted population of cells that will persist as you have heard from the scientists at CRISPR discussed here just a moment ago. So we're very much encouraged with the signals that we're seeing in terms of path of focus. We hope
that that continues and we'll continue monitoring very closely.
Yeah. Thank you, Alina. And I will just add, June, to your question. You heard from
whom we have discussed today has a durable complete response. And our experience with the autologous constructs is that majority of patients who go into complete remission are staying in complete remission long term. We expect and I'll just extrapolate it. We expect that we're going to see the same thing here. And so we're greatly encouraged about the potential here.
We really think, again, this is the next generation, this is the future for our patients. That's why we're participating in this important study.
Thank you, Doctor. Maguirek. And, Erlina, to the second part of your question around, the ICANN, so I think we Erlina disclosed them as well as providing you on CTX-one hundred and twenty and CTX-one hundred and thirty.
Due to brevity of time, this does
Hi, guys. Thanks for taking my questions and congrats on the data as well. So one of the questions in dose level 2 who may have relapsed is associated with different levels of
Let me turn it over to Evelina to answer that question.
Biological course that was both the result of lymphodepletion, HHP6, encephalitis and ICANS. And as Sam mentioned, we're going to continue evaluating as we treat patients. And I just wanted to point out that all the patients treated at dose level 3 had no CRS and no
Yes. Thank you, Elena. And I will just add, June, to your questions. You heard from Doctor. McGurk.
I really think as we look at the early responses here, these AOPs and build the chassis for the next generation of Allergan XL therapies, which can have impact on these patients that have very limited options. I think on the safety as well, I think we see a relatively acceptable safety profile and we are full steam ahead on these trials and we look forward.
Due to brevity of time, this does conclude our question and answer session as well as our conference call for this morning. You may now disconnect your lines and everyone have a good day.