Q3 2022 Sangamo Therapeutics Inc Earnings Call
Yeah.
The conference will begin shortly to raise your hand during Q&A you can dial star one one.
[music].
Good day and thank you for standing by welcome to the Sangamo Therapeutics third quarter earnings call. At this time all participants are in a listen only mode. After the speaker's presentation there'll be a question and answer session.
Ask a question during this session you will need to press star one one on your telephone you will then hear an automated message advising that your hand is raised.
Please be advised that this call is being recorded.
I will now turn it over to Louise Wilkie head of corporate Communications and Investor Relations. Please go ahead.
Good afternoon.
Okay, Thank Mike Vice President Investor Relations and corporate communications.
Thank you for joining us on the call today.
On this call are several members of my executive leadership team, including Sandy Macrae, Chief Executive Officer, Mark Mcclung, Chief operating officer, but you should do a Barber Chief Financial Officer, Jason <unk>, Chief Scientific Officer, Nathalie Dubois, Stringfellow, Chief Development Officer, and Bettina Cockroft Chief met.
All of them.
Slides from our corporate presentation can be found on our website sangamo dot com under the investors and media section on the events and presentations page.
This call includes forward looking statements regarding <unk> current expectations. These statements include but are not limited to statements relating to the therapeutic and commercial potential of our product candidates.
The anticipated plans and timelines of Sangamo collaborators for initiating in conducting clinical trials testing and screening patients and presenting clinical data.
Most of our product candidates.
Advanced with a preclinical programs to the clinic.
Our investment focus and the sufficiency of our resources.
2022 financial guidance upcoming catalysts and other statements that are not historical facts actual results may differ materially from what we discuss today. These.
These statements are subject to certain risks and uncertainties are discussed in our filings with the SEC specifically in our annual report on Form 10-K for the fiscal year ended December 31 2021.
Supplemented by our quarterly reports on Form 10-Q for the fiscal quarter ended Sept.
Timber 32022.
Forward looking statements stated today are made as of this date.
No duty to update such information, except as required by law.
This call will discuss our non-GAAP operating expenses reconciliation of this measure to our GAAP operating expenses can be found in today's press release, which is available on our website.
Now I'd like to turn the call over to our CEO Sandy Macrae.
Thank you Luis and good afternoon to everyone on the call.
<unk> will continue to pursue scientific innovation and development excellence and the third quarter as we progressed our mission turning groundbreaking science into potentially transformative genomic medicines for patients.
I am pleased with the momentum we are achieving across our programs with our clinical data continuing to demonstrate the strength of our pipeline.
Regarding our wholly owned Fabry disease program, we were excited to present additional preliminary data from our phase <unk> study of three separate medical Congresses since the last call.
We continue to be encouraged by the promising data generated to date, showing sustained and elevate yourself per galley activity along with a favorable safety profile as of the last data cut in July .
These data demonstrate the potential of this therapy to replace the current standard of care and provides the potential for onetime dosing alternatives rather than a lifetime of ERP infusions.
I'm also pleased to share that the study has moved into the dose expansion piece and we've begun dosing patients, including the first female patients in this study.
We continue to make progress in our wholly owned sickle cell program this quarter dosing the six patients in the phase two precision study.
This is the second patient to be dosed with their product candidates manufactured using improved methods.
Had reopened recruitment trial sites resumed enrollment in September dosing expected to resume shortly their assumption of this study is an important milestone as it takes us with our partner Pfizer one step closer to getting this potential treatment to patients in an area of significant unmet medical need.
I'm proud of the team for the advance since you continue to make staying true to our strategic focus on our research and development capabilities.
Each incremental update brings us closer to a goal of creating transformative medicines for patience, while creating longterm value for shareholders.
I'd like to turn the call over to her headed development, Natalie who will discuss the data from a clinical programs in more detail.
Natalie.
Thank you Sandy and good afternoon to everyone on the call.
We had several promising update in the third quarter and are pleased with the progress being made across our clinical program.
In February 5th station with withdrawing some enzyme replacement therapy in the faith wanted to start clinical study.
<unk> <unk> <unk> <unk> or S. T 920, I only one gene therapy product candidate for the treatment of February disease.
This might the fifth and final patient in the dose escalation state started this study on E. R. T. Two has been withdrawn from the I T.
All subject report they are not experiencing changes in symptom and not require assumption of the I T.
After receiving an endorsement from the <unk> when to study safety monitoring committee to progress to the dose expensive.
Five station has been dose in this expansion phase at the five G 13 be cheaper cake dose levels, including the first two female patients in the study.
Mentum also continue with additional patient currently screening and waiting dosing, including more female patients.
Since the last call. We we're proud to present updated preliminary February data at three medical conferences.
August we presented data at the society of the study of inborn errors of metabolic <unk> S. S. I M annual meeting in Freeburg, Germany.
Presenting data on five patients as of February 14 data capped.
Last month, we provided further updates at the 29th annual Congress of the European Society of gene in South therapy, ESG C. T in Edinburgh, Scotland as well as the National organization for rare disorders Nord Conference in Washington D C.
At both this event, we presented data as of July 21st 2022, presenting for the first time data across all sorts dose cohort in the dose escalation phase.
All nine treated patients exhibited sustain elevated alpha.
Activity wrenching from nearly twofold 230 fold of men normal for almost two years of follow up in the longest treated patients.
All patients exhibited above normal of alpha galli activity by five weeks after dosing and importantly, also patient and long term follow up maintain elevated alpha gal levels for one year or more.
We remain encouraged to see their ability of effect for this month of time also of note for patients have undergone <unk> withdrawal as the kind of date and continued to maintain elevated alpha gal activity.
Up to 28 weeks post withdrawal.
The two subjects, we substantially higher elevation in class My life would you be three pre treatment she'll, a 40% and 55% reduction from baseline in life that she'd be three level, respectively. After S. Two 920 doses.
Importantly, S. T 920 continued to be generally well tolerated in the nine treated patients with no treatment related adverse event higher than great one except for one great to Pikes yeah.
No treatment related serious adverse events were recorded.
No no subtract I've been treated with steroids, either prophylactically or reactively and I've been known liver enzyme elevation requiring treatment.
Since our last call additional side in this global study has been opened including the first in Asia Pacific and we're actively preparing additional new site.
You can expect further clinical updates from the start study, including the first data from the expansion cohort in the first half of 2023.
We continued to activity plan for a potential face, we study and gauging with the health authority patient advocacy group and investigators.
In the phase one two precision study of <unk> I think stinker nucleus editors cell therapy candidate for the treatment of sickle cell disease.
Wholly owned by Sangamo, we dose of six patient. This is the second patient two has been dose with a product candidate manufactured using improved method shown in internal experiment to increase the number of long term progenitor cells in the final product.
We are pleased to continue to be making progress in the face went to study and look forward for providing additional update at an appropriate time.
Ah Sunday mentioned earlier on August 24th So FTA granted Sanga most requests for originality of medicine advanced therapy or Matt This nation for <unk>.
I'm not designation is granted to retinal if medicine therapies intended to treat modify reverse or pure serious condition for which preliminary clinical evidence indicates that this medicine as a potential to address an unmet medical needs.
The automat designation includes all the benefit of the fast track and breakthrough therapy designation programs, including early interaction with F. D. A.
Furthermore, we were accepted to participate in a poster presentation at the upcoming 64 American Society of Hematology Ash annual meeting and Exposition, taking place in New Orleans, Louisiana.
Number 10 13 2022.
We look forward to presenting updated clinical data then.
Finally phase three study design, enabling activities and manufacturing readiness in progress.
Progress continue in the face one too steadfast study R. <unk> 200 car to you break cell therapy candidate for the prevention of immune mediated rejection and HL eight two mismatch kidney transplantation from a living donor.
The second station was dose in late September and I'm pleased to report that the product candidate continued to be generally well tolerated in both patient.
To control patient I've been enroll in transplanted demonstrating ongoing interest in this study we will provide further guidance on timing for dosing of the third patients when the transplant has been schedule and we have confirmed a potential dosing thing.
Finally regarding the phase III find trial evaluating <unk> and invest tingle investigation or gene therapy for <unk>.
We jointly announced with our partner Pfizer.
The trial as we open recruitment.
Trial sites resume enrollment in September and dosing is expected to resume shortly.
A pivotal read out is expected in the first half of 2024.
In addition, with our partner Pfizer, we've been accepted to participate in a poster presentation at Ash December 10th 213 2022 provide.
Providing updated clinical data on the face went to <unk> study, we look forward to sharing updated data in December .
I will now turn the call over to our Chief Scientific officer for updates on our preclinical research program.
Jason.
Thank you and good afternoon, everyone.
I'm pleased to report that we continue to leverage payment was cutting edge genomic engineering console therapy platforms to advance both wholly owned and order programs toward the clinic.
We're also making remarkable progress and are preclinical programs as well as expanding our proprietary genomic engineering tool kit and developing novel a decaf.
More effective therapeutically relevant delivery of these tools.
Recyclable team had multiple presentations at this year's European Society of G cell therapy annual conference.
Including work describing the use of our proprietary thing finger nucleated platform to engineer allogeneic healthy donor regulatory <unk> and apologists visa Bill.
Additionally, data from preclinical studies performed with our collaborators at the Massachusetts Institute of Technology <unk> Institute represented at the International pre on 2022 conference and go to that in Germany. The September .
We were thrilled to hear strong feedback from key opinion leaders at the conference we express their excitement Patel.
Potential for patients need.
This work demonstrated the ability of famous engineered finger protein transcription factors to specifically reduced prion protein expression in the brain is significantly extend the survival in a mouse model.
We look forward to providing updates on this and other programs in the future.
Will now turning the call over to our Chief Financial Officer.
For an overview of the financial results.
Sure.
Thank you, Jason and good afternoon, everyone.
A detailed financial so for this quarter are available in the press release issued this afternoon, which can be found on our website.
We ended the quarter with approximately 350 million cash cash equivalents and marketable securities, which represents and Netflix client of $14 million from the prior quarter.
This reflects a proactive approach to both prudent Kappa management and balance capital races. In these uncertain market and speaks to the continued in methane crest in our company.
We've reached approximately 75 million and Netflix <unk>.
At the market offering program since the beginning of the year.
We continue to focus our investments in three key areas.
Advancement of a clinical program, including separate sickle cell antiques 200.
Progression of a preclinical cocky break emptiness pipeline and optimization of our in house manufacturing capabilities.
Turning to our 2022, who you garden <unk>.
We expect a full year non-GAAP operating expenses to be lower than previously guidance and land between 280 million to $490 million.
This screen exclude the estimated non-cash stock based compensation expense of approximately $35 million.
We expect a significant portion of our operating expenses to be invested in the continued advancement of <unk> programs.
Clothing February 50 planning activities.
One two activities <unk> 200, and pre clinical work in court directs and genome engineering indications in the central nervous system.
I will now turn the call back to Sandy for closing remarks.
Patricia.
We are pleased with our achievements during this quarter and the progress with major crosser clinical and preclinical pipeline.
We are a clinical stage medicine company building momentum with a <unk> clinical execution.
<unk> manufacturing.
The progress this quarter, Texas, one step closer to lifting potentially transformative therapy for patients in need for creating longterm value for shareholders.
I'm so proud of our team here to assign will who work tirelessly to advance our programs intaglio term mission.
We are delighted with the postal momentum and look forward to seeing you in the near term with several expected milestones, including presentation stash of <unk> data and sickle cell disease.
Presentation of Ash of updated phase one two decent hemophilia.
Additional fees one <unk> in the first half of 2023, including additional ERP withdrawal data and the first data from the expansion fees.
As soon as the third patient and the steadfast please one to study.
At this time, we would like to open up for questions.
Operator, please open the line for questions.
Thank you at this time, we will conduct a question and answer session. As a reminder to ask a question you will need to press star one one on your telephone and wait for your name to be announced please standby as they compile the Q&A roster.
Our first question comes from Genoa, Wang with Barclays. Kennedy Your line is open.
Thank you for taking my questions I have two questions. The first one is a sickle cell dash and then we all saw the abstract released earlier today. So it seems like a place holder data cut off may 3rd.
At the full data presentation <unk> locations at Ash and then my second question is that February program.
So regarding the expansion cohort data in first half twenty-three sure. The five G data or will we see more patients.
Data up your current angle two to five patient and also what type of data you'll be sharing would that be mainly biomarker data like Africa and a license.
What kind of a clinical profile.
Go to move to the next step.
Good afternoon, and thank you for your question, So Oh, sorry, I understand you're asking a question that cycle and a question of February So I'm Gonna pass <unk> to Nutley, and then the <unk> will cover the fabric question badly.
Yeah, Hi, good afternoon Gina.
So patient five in the trial was Josie may 2022, and it was the first patient to receive a product candidate manufacturing use using the input process. We expect to share early preliminary data from the station at Ash in December .
Yeah, a poster presentation, along with an update of the first spot patient dose.
So Gina thank you for the question around February . So we are as you as you are.
See we're very excited actually to be sharing that we have now those five patients in the expansion cohort.
Brings us to a total of 14 patience dose in the <unk>.
The study of which if you do the math it means we have seven patients treated at the highest level.
920 has the potential to July .
Alternatively, the current standard of care.
We will be sharing we pointed out in the first half of 2023 data from <unk>.
Additional data from the those escalation as well as the early data from the expansion cohort patients and this will include by market data as well as additional clinical data the exact nature, which we will share.
First half so in early 2023.
Thank you.
Please stand by for the next question.
Our next question comes from Luca <unk> with our B C. Go ahead Luka.
Oh perfect. Thank you. This is Lisa Luka. Thank you so much for taking my question.
Two on Saturday for you today.
First of all the data just wanted to follow up on the maybe data presented at yesterday's D. T last month I think we noticed there was at least one patient.
Who was withdrawn from your teeth, but subsequently had an increase in the plasma Alpha Galaxy Tiffany after the withdrawal. So I was just wondering how we should Rationalises response, given you would expect that the alpha <unk> levels to drop after the E. R T withdrawal.
And secondly is on the phase three planning you know <unk>.
<unk> reiterated today that you're facing planning is underway I'm. Just wondering if you have had discussions with the F. D. A here.
<unk> could look like what would be the primary endpoint and what you would need to show in terms of the ability given this visit gene therapy approach. Thank you.
So thank you for your questions and thank you for.
[noise] such attention to our <unk> presentation, So I'll I'll I'll help I'm Gonna pass this over to Pacino, but I'll help her little was the second part.
We've had <unk>.
We've had lots of time to plan and sing to both the phase III study, we've spoken to the agency and will share the details without starting a new this time so.
Forgive me cause I know I'm interested everyone is in the details of that study.
Tina could you answer the first part absolutely and thank you. So much for that question. Some patients on E. R. T who have withdrawn from E. R. T. You asked about the Alpha go a uhm activity expression, we do expect alpha galle expression to be sustained over time.
Time, and that's what we are seeing not only in patients who were naive or pseudo naive, but also patients on E. R. T who was withdrawing from <unk> that is the objective of our therapy and in fact, we believe that S. T 920 has that potential to provide an alternative to.
The current standard of care of E. R T.
If you were referring to Plasmolyse GB three levels. These specific patients are all doing well.
<unk> G. B three remains within the range seen in patients on the I T. B investigators are happy with the data patient.
Patients remain of T. R. T I hope that addresses your question.
Mmm.
It does thank you.
And I would encourage you to remember the the shoes.
Cutting edge clinical sowing seeds of the first patients that we've.
<unk> data on with this treatment with the withdrawal of <unk>. The early data that you've seen this with the lowest dose response, we hope we plan to be withdrawing <unk> the.
The dose we've chosen to go forward into phase three.
Sure patients withdrawn from <unk>.
The other thing I would I would ask you to think about is even for patients only F. T. From we added an urgent need therapy. This shoot additional benefit and I think this is sometimes overlooked patients had been on New York T for a long time <unk> the gene therapy reported feeling better that they were sweating.
And there was evidence of stabilization or even improvement of the <unk> function really speaking to the power of the Smith.
Please stand by for our next question.
Our next question comes from Nicole Jeremy now with Trust your line is open.
Hi, This is Alexandra Nicole.
The card to Red platform would you consider outlicensing or engaging in other collaborations with big pharma and if so.
Some color on the types of partnerships that you consider that might work best for say goodbye.
And related what are your thoughts on big pharma dropping.
Two programs and services.
Is it auto immunity and does this change or does it paved the way for Carcieri eggs.
Space.
Mark you you run a business development group, we just wanted to talk to them.
Sure. Thanks for your question you know so.
Obviously, we're excited with the progress, we're making with key rags, where we believe we're the first company to have those probation as we've communicated earlier our commitment is to continue to move forward with that studying and drive it towards a.
Proof of concept you know if companies do approach us similar to what we've done in the past if if they can bring resources knowhow and.
And funding actually to accelerate the patients accelerate the the therapy to patients faster than weekend, we will always evaluate that but.
But we're not commenting on any current conversations that we might be having in that regard.
Thanks, and also on that we've seen in the news some big pharma dropping IELTS you've platform. Some have you seen.
M S N.
Do you have any comments.
Jason R. Two scientific of suggestion would you be able to comment on I'll tune in where we fit in amongst the range of T Rex programs.
Thank you Sir Thanks for the question the latest news that I'm aware of on a form of dropping in Iowa to program was related to the use of oil too apology, which is a different approach then some of the <unk>.
That are being explored two and half tea read biology.
But.
Whichever the case is it's important to point out that while <unk> can be used to expand regulatory T cells and do offer with your potential or therapeutic benefit an auto immune disease, it's a fundamentally different approach because it relies on system.
<unk> delivery of that oil too and increasing.
Tori T cells, and and the entire body of the patient.
The approach, we're taking with core regulatory T cells is the target.
The regulatory biology itself using American image receptor to the relevant tissue. So that's a much different proposition where the.
Kind of a new regulation that the regulatory T cells afford is directed in a.
<unk> had a specific and very potent way towards the tissue, where the auto immune to pop mythologies Kurt Oro.
<unk> the aisle too new teams. Despite best efforts still do have some reactivity with <unk> and K T self and so this is confounding any interpretations of the effectiveness of those because by <unk> also hitting up after <unk> one runs the risk of.
Surely exacerbating.
Mean disease.
Okay. Thank you Jason for the color.
Please stand by for the next question.
Our next question comes from Jan and do with Wells Fargo. Your line is open.
Hi, Thanks for taking our questions a question <unk> and a question a sickle cell program. So for for the families. Yes, GCT presentation I think you alluded to this earlier.
And it looks like the lifestyle GB sweet level of increase in patients who discontinued.
T, but says that data cut off is rather early.
July 21, 2022, it's been quite a few months since that data cut off and I think you mentioned and nobody has a resume E. R. T. So I was just wondering.
Could we take that as meaning the patients have somehow stabilized they're likely be three levels and could you also made us what's the criteria for resuming T X.
Thank you. Thank you for your question is always between US can you give us some clarification on that please.
For sure. Thank you for that question. So we were very pleased to present the latest February data ESG C T.
We I can confirm that we now have five patients who are of E. R. T and that none of them have resumed ER T therapy investigators are happy with the data they are seeing including the plasma nicer GB three data base of seeing.
And in terms of criteria for resumption of ER tea that is really dependent on a series of biomarker in clinical data and ultimately a decision to invest the data with the patient and so far.
Those patients are doing well.
I think that's the most important thing that it's an investigator cole the patients are doing well in there for the investigators comforted to keep them off under the levels you've seen are similar to other.
Other patients all New York to boot up for their lives with G. P. Three indeed.
Got it that's a very good to hear for the <unk> program.
Talks about five three design and manufacturing redness or underway I was wondering how much of those progress.
Has been informed by what you saw in patient number five.
The the patient on the new manufacturing product and also do you plan to pursue this program by yourself or or how urgent is it to to find a partner for this program. Thank you.
Natalie can you answer that one.
We're currently thank you for your question with currently focused on completing the phase one to study and progressing phase III, enabling activities. We're really excited to have this program and a hand and we're planning to take this program, Florida, assuming the data continued to support a phase III program. So the second cohort those.
Next station that have.
Hi, sorry, the next patient that have improved manufacturing will be very informative to see if we if we have a body of evidence to to continue to two phase III, but we're planning ahead and you'll have to wait for ash poster to see a little bit more data.
Got it in partnership.
Initiative.
We.
<unk> intention is always to do the best for the program.
<unk> gathers the data and so we won't really see the few dataset from the new improved process and two first quarter first half of next year.
The appointment will meet the necessary decision, we look at is whether.
The waiter or for their partner does it's getting a successful mentioned to patients that's the important thing.
Got it thank you.
Please stand by for our next question.
Our next question comes from Greg Harrison with Bank of America. Your line is now open.
Hi, there. This is Mary counter great. Thanks for taking my question I guess regarding February is your purchases free trial could you comment on the physician in teaching I'd be interested in a gene therapy for February and the impact of Iraqi freedom with population, maybe what is clinically.
Thank you.
Your <unk>. Your line is is hard to follow could I ask you just to repeat the the.
A truck so the question please.
Yeah, absolutely so as your purchases two child could you comment on the physician.
The interest in a cheeseburger, please and also the impact of D. R T freedom.
Okay, <unk> I think this might be one for Ya.
Thanks for the question.
So first of all I mean, just as a general rule.
We actually engage with advocacy very early actually prior to phase one and work with them to provide input into our protocol designs, but also to better understand the patients. We also bring patients into so to speak to us to share their disease, so that our.
And researchers and developers actually can really understand what the patient requirements are so so that's something that we're particularly proud of and I think based on my experience back about does incredibly well.
You know in terms of.
Next steps will will provide next steps you know at the at the appropriate time, but I think that.
I think that we're making great progress with the program I think it's important that we finalize the deal.
Details of that and then we can provide the appropriate guidance to you later.
Mmm.
Thank you.
Please stand by for our next question.
Our next question is from Kelly Breza with Stifel. Your line is now open.
Hi, Thanks for taking the call. My question. This is categories for Ben Burnett Ah.
I just had one quick question actually regarding some of your preclinical programs and brain disorders, such as the crayons.
Does the Tao therapies Huntington I was wondering if you could talk about these a little bit in more detail and and just maybe one you'll be seeing these maturing into the clinical phase. Thank you.
Thank you for your question, we would love to share more about them. Unfortunately, <unk> and these partnerships. The communication on those is controlled by the partners. So probably would I would want to reassure you as the people who run the table here sit on steering committees development committees research committees with the <unk>.
And they're moving forward and so we we almost have a a hidden early portfolio that is in our in our park tons, they're passionate about them. They loved the signs of <unk> and we hope to be able to say more sutent, let's see let's see get through the milestones in towards the clinic.
[noise], we do.
Did you did you all sorts of <unk>.
Jason I know how excited you are but the prion.
Results can you talk to or not because I think is a remarkable piece of science.
Yeah. Thanks Sandi.
One of our our scientists Brian Zeisler from from Sangamo presented at <unk> 2020 earlier. This year data that <unk> is generated in collaboration with the broad Institute at the Massachusetts Institute of Technology, where are we were studying the ability.
<unk> of our as a finger transcription over oppressors to be delivered to the brain of of mice in a in a mouse model of prion disease and repress the expression of prion protein and thus prevent the spread prion disease and the the data.
[noise] that was generated there was really.
Really remarkable compared to control and compare to other approaches that we've known have been tried in a similar models such as aso's. The the ability of the finger transcriptional oppressors, two potently repress prion protein X.
Oppression and dramatically extend the life.
Of the mice in the smells model prion disease were very impressive and several key opinion leaders in the field international experts in Creon disease made comments about how impressed they were with the data.
There was even one comment that it was the most exciting data of the conference. So this is a program that we're very excited about with a wholly owned the internal program and we are exploring moving it forward into humans as as soon as we can of course there are additional complications this was mouse data and now we're.
Dealing with the the next steps to put together a package that would allow us to move it forward into.
For testing in humans if possible.
Great. Thanks.
Please stand by for our next question.
Our next question comes from <unk> with Cohen. Your line is now open.
Good afternoon, guys. Thanks for taking the question I wanted to ask about the profile of the female fabry patients.
Unrolled are they are they under four E. R T dose and I'm wondering about their kidney versus potential cardiac involvement or aspects of their disease and further and this expansion cohort target number of female patients that you're looking to involve 12.
<unk> and then I've got a follow up.
Thank you for your questions yet, it's exciting where we've moved into female patients, but you know can you give any more color on them.
Yes, so for sure I mean, what we have done in terms of the patient characteristics you can see that data, but there's a lot of detail and R. E. S. G. T. Five so we have patience with them various characteristics. We do plan in the protocol allows up to 30 patients to be those as part of the expansion.
Face and that includes both males and females. It also includes Reno cohort carjack cohort. So we're not dining at this moment us to the details.
How many patients we have or plan to have in those specific cohorts, but will be making an update in early 2023 around that but you know I hope I'm not much speaking here, but there are five different cohort potentially five different cohorts, that's right and I think we presented that it ESG C T.
We have one cohort with.
Uhm antibody positive patients another one with antibody negative patients. Another cohort has female patients. Another one is Reno cohort and another one is a cardiac cohort and of course. These latter two cohorts can include both male and female so so the females could be.
That cohort plus females.
With other forms of the disease does that help.
It does that's helpful. Thank you and then can you can you guys comment on.
Where you stand on CMC for a commercial product for the potential launch that tip at all.
Especially on things that have.
Tripped up other spot.
Sponsors.
Assays that.
<unk> Clinton C assays, all that sort of thing.
So we we take the part of development as seriously as we do the clinical piece and it was a <unk>.
<unk> I'm glad <unk> cause I knew the clinical piece get some most airtime and excitement, but the only way to develop these medicine is paying attention to the C. M C and the manufacturing and we're we're very under control we have that's the thing.
Setup to go for both the phase three and commercial.
Got it and can I squeeze one last question.
You elaborate sandy a little bit on the biopsy schedule for patients.
<unk>.
Bettina.
Absolutely so for patients in the expansion.
Cohort well first of all we do have two patients from cohort for who are naive patients for whom we have collected.
Collected biopsy data.
And we those will be earliest patients with kidney biopsy data we plan to share that information in early 2023, and we are collecting kidney biopsy is also for for patients who are naive or pseudo naive.
In the expansion cohort data will will read out as as the study progressive we're very grateful to the patience for volunteering.
She's kidney biopsy is are not a minor thing to have and to top.
<unk> the patient have booster entry biopsy and then to come back later for him. After one reminds us of our gratitude to patients and what they do to make these struck development possible.
12 80.
Five seats.
These are six months biopsy okay.
Got it thank you.
Please stand by for our next question.
Our next question comes from Patrick <unk> with H C Wainwright and <unk>. Your line is now open.
Hi, Good afternoon, just a couple of follow ups just the first one is on the Fabry program I'm wondering if you can discuss in more detail the safety and Tolerability profile that you've seen so far in the program and if you can talk more specifically what it is about it that has enabled you to avoid some of the side effects and adverse events that we've seen with some of these other programs.
Space and to what extent could be acquiring will be applied the current or future gene therapy, you're editing programs and development from the platform.
Thanks for your question Patina, you don't really have much to say about the safety profile, which is good [laughter]. So thank you Sandy S. So on the safety and Tolerability side as he'd seen we are doing extremely well patients are doing extremely well we have not needed.
To treat any of the patients with steroids.
Reactively patients are not treated with steroids prophylactically, we we continue to monitor patients and as I mentioned earlier, we now have seven patients both at the highest those cohort so.
It's a question of monitoring these places in the next patient and I think what the clinical trial team has really been doing well is paying a lot of attention to the details around.
Education around the infusion schedule. So we're very pleased with with the progress on the safety and Tolerability side.
And we've used a V six <unk> across the M. P. S trials for hemophilia trials and I've fabrics, and so we've got quite a broad.
Patient experience and it is remarkably well tolerated, even if only fee 13 the the.
The only thing that we saw or we we have seen in the hemophilia trials.
His occasional L. S T events.
Our resolved quickly and easily with steroids. What's interesting is that we haven't seen any of these thus far in the sound pretty strong and the the question must be <unk>.
The the truth is that we don't have enough experience shifting of patients followed to understand why is it.
Why is it the cargo seems to make some kind of difference in this.
Yeah.
Helpful. And then just call me on the preclinical pipeline.
Following up on some of the discussion earlier, there's many preclinical programs in the area of the car to your lifestyle therapies for auto immune disorder, and as well as the United anchor neurological disease, and many indications of internal and external your partner. So I'm wondering if you could frame for us which program or programs do you think are perhaps not as well appreciate it.
Some of these more advanced clinical programs and when when we can expect from the next updates on those for somebody who's under appreciated programs.
Mmm.
Thank you, Jason you're very excited by the car to write programs on the C. N. S. Can you can you say, which appears which ones you, particularly with wants to highlight.
Well thank sandi.
In the court T. Reg space, we've we've discussed the programs that that we're moving forward in the preclinical space in multiple sclerosis, and inflammatory bowel disease, we believe that both of those areas.
Our our our great opportunities for the therapeutic use of T Rex and really I'm I'm excited about the the advancement of both of those regulatory T cells offer the potential to transform the treatment of auto immune disease by.
Targeting immune regulation to us to a specific tissue for example in the in the CNS for multiple sclerosis, and the ability to offer a a durable long term benefit to patients rather than constantly being treated with it with the medication for that.
A lifetime of a disease. So it's really a you know the way we view the court T. Reg platform is something that has the potential to transform the treatment of auto immune disease, and we're very excited about those programs moving forward.
In the CNS space I'd say, what what's most exciting is you know is the diversity of of the pipeline.
Have our the finger transfer transcriptional regulators, which just as a reminder, can either upregulate or Downregulate G and those are being used in collaboration with Biogen and our collaboration with Novartis in in Neurodegenerative diseases.
Is a developmental.
Developmental diseases, respectively. We're very excited about the progress that's being made there are partners are really excited.
I think the fact that those partnerships and those those companies came to Sangamo acknowledges the amazing tool kit that we have.
Also advancing our own internal programs and prion with highlighted in we have several other non disclose programs that we're looking forward to share data with at the appropriate time.
And on top of that an area that I'm, probably most excited about in the last year is the advances that we've made in our a V. Cassidy engineering work. So we have a very I'm healthy effort in developing new a V caps is that can deliver our.
Finger tools to the CNS, either by direct injection or by by the blood through go across the blood brain barrier and we've made some some remarkable advances there some of that data was presented at a S. G. C. T earlier this year and the teams that are doing those that work.
Those directed evolution studies to identify new cabinets that are even more effective.
Have been continuing to generate exciting results with those cast is it's gonna open up even more more space indication space to go after more and more diseases. It says at the moment really the zinc fingers are working amazingly well and it's it's really a matter of getting.
Getting those tools to the the right cells in the body and getting the coverage that's necessary and that's with a the new a V cabinets that we're developing will afford so we're super excited about that work as well.
That's helpful. Thank you very much.
At this time I would now like to turn it back to Luis Lucky for closing remarks.
Mmm.
Thank you. Thanks, once again for joining us today in preference question. As a reminder, you can you can access the earnings release and presentation on Investor Relations section of my website. We look forward to keep you updated on all future developments. Thank you.
Thank you for your participation in today's conference. This does conclude the program you may now disconnect.
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