Q3 2020 Sangamo Therapeutics Inc Earnings Call

Ladies and gentlemen, thank you for standing by and welcome to Sangamo Therapeutics Q3, 2020 webcast at.

Operator: Ladies and gentlemen, thank you for standing by, and welcome to Sangamo Therapeutics' Q3 2020 webcast. At this time, all participants are in a listen-only mode.

At this time all participants are in listen only mode. After the speaker presentation. There will be a question and answer session to ask a question. During the session you will need to press star one on your telephone.

Operator: After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference may be recorded. Should you require any further assistance, please press star zero. I would now like to hand the conference over to your host, Head of Corporate Communications, Erin Feingold. Madam, please go ahead.

Please be advised that todays conference maybe recorded.

If you require any further assistance please press star zero.

I would now like to hand, the conference over to your host head of corporate communications and find goat Madam. Please go ahead.

Erin Feingold: Good afternoon, and thank you for joining us today. With me on this call are several members of the Sangamo Executive Leadership Team, including Sandy Macrae, Chief Executive Officer, Sung Lee, Chief Financial Officer, Mark McClung, Chief Business Officer, Jason Fontenot, Interim Head of Research, and Bettina Cockroft, Chief Medical Officer. Slides from our corporate presentation can be found on our website, sangamo.com, under the investors and media section in the events and presentations section.

Good afternoon, and thank you for joining us today.

With me. This afternoon on this call are suffering numbers assessing demo executive leadership team, including Sandy Macrae, Chief Executive Officer sung Lee Chief Financial Officer, Mark Smith, Chief Business Officer, Jason Oh, No interim head of research and Bettina Cockrell Chief.

Medical Officer.

Slide from our corporate presentation can be found at our website <unk> dot com under the investors and media section in the events and presentations page.

Erin Feingold: This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to our R&D pipeline, our ability to develop, obtain regulatory approvals for, and commercialize therapies to treat certain diseases, and the timing, availability, and cost of such therapies, plans and timelines for Sangamo and our collaborators to conduct clinical trials and share clinical data and the potential for these data to demonstrate clinical benefits to patients, and the potential to use certain technologies to develop our therapies. Our collaboration strategy and the potential to earn milestone payments and royalties from our collaborations, and the timing of receiving such payments and royalties. Plans and Timelines for Building and Opening Manufacturing Facilities, the Effects of the Evolving COVID-19 Pandemic, our Expectations regarding our Financial Performance and Resources, and Other Statements that are Not Historical Facts However, actual results may differ substantially from what we discussed today.

This call includes forward looking statements regarding single most current expectation.

These statements include but are not limited to statements relating to our R&D pipeline.

Our ability to develop obtain regulatory approvals for and commercialize therapies to treat certain diseases, and the timing availability and cost of such therapies.

And timelines for Sangamo, and our collaborators to conduct clinical trials and share clinical data and the potential for these data to demonstrate clinical benefits to patients.

The potential to use certain technologies to develop our therapies.

Our collaboration strategy and the potential to earn milestone payments and royalties from our collaboration and the timing of receiving such payments and royalties.

Plans and timelines for building and opening manufacturing facilities.

The effects of the evolving 'cause it 19 pandemic.

Our expectations regarding our financial performance and resources and other statements that are not historical facts.

Actual results may differ substantially from what we discuss today.

Erin Feingold: In addition, these statements are not guarantees of future performance and are subject to certain risks and uncertainties that are discussed in documents that we file with the Securities and Exchange Commission, specifically in our quarterly report on Form 10-Q for the quarter-ended September 30, 2020. The forward-looking statements stated today are made as of this date, and we undertake no duty to update such information except as required under applicable law. On this call, we discuss a non-GAAP financial measure. We believe this measure is helpful in understanding our past financial performance and our potential future results. This is not meant to be considered in isolation or as a substitute for the comparable gap measure.

In addition, these statements are not guarantees of future performance and are subject to certain risks and uncertainties that are discussed in documents that we file with the securities and Exchange Commission specifically in our quarterly report on form 10-Q for the quarter ended September Thirtyth 2020.

The forward looking statements dated today are made I suppose this date and we undertake no duty to update such information, except as required under applicable law.

On this call we discussed a non-GAAP financial measure we believe this measure is helpful in understanding our past financial performance and our potential future results.

This is not nice to be considered in isolation or as a substitute for the comparable GAAP measures the comparable GAAP measure and reconciliations of GAAP to the non-GAAP measures discussed on this call are included in today's press release, which is available on our website.

Erin Feingold: The comparable gap measure and reconciliations of gap to the non-gap measure discussed on this call are included 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, Aaron, and good afternoon to everyone on the call. This quarter, we advanced our R&D activities as we continue to adapt to the conditions brought on by the evolving COVID-19 pandemic. We are moving forward on clinical execution, and we are optimistic about our plans to continue to dose patients and initiate new trials. We have also completed our research activities associated with our ALS collaboration with Pfizer, and are continuing to move our research projects with Biogen and Novartis forward, and are progressing our work with our other partners. Pfizer dosed the first participant in the Phase 3 Affine Study of Girotopogene Phytoparvovic, or SB525, our investigational gene therapy for Haemophilia A patients. This event triggered a $30 million milestone achievement for Sangamo, which we expect to receive in the current quarter, further strengthening our cash position.

Now I'd like to turn the call over to our CEO Sandy Macrae.

Thank you and good afternoon to everyone in the call.

This quarter, we advanced our R&D activities as we continue to adapt to the conditions, Brooklyn, but evolving coupons to consumers.

We are moving forward on clinical execution and we're optimistic we're plans can you continue to dose patients and initiate neutrons.

We also completed our research activities associated with our email list country collaboration with Pfizer.

And are continuing to do for research projects with Biogen and the boxers forward and.

And are progressing I work with other partners.

Pfizer has two is the first participant in the phase three a fine study about you talk could you speak to poverty flourish speaks five to five or investigational gene therapy for hemophilia a patients.

This has been triggered a $13 million milestone achievement for cycle, which we expect to receive during the current quarter further strengthening our cash position.

Alexander D. Macrae: Pfizer previously communicated that they expect a pivotal data readout from the AFIND study in 2022. During its mid-September investor day, Pfizer provided an update from the Phase 1-2 ALTA study showing encouraging data regarding tolerability, clinically meaningful factor levels, bleeding rates, and factor use in the highest-dose cohort up to 85 weeks in the longest-treated patients. Pfizer and Sangamo believe that these data support a potentially differentiated haemophilia A gene therapy product candidate.

Pfizer previously communicated that they expect a pivotal data readout from the a flame study in 2022.

During the mid September Investor Day, Pfizer completed an uptick from the phase one two ultra study showing encouraging due to the garden tolerability clinically meaningful factor liftings bleeding rates and fun to use in the high schools cohort dropped to 85 weeks in the longest.

We should patients.

Pfizer insight and we believe that these data support to potentially differentiated hemophilia a gene therapy product candidate.

In August and September in close collaboration with principal investigators monitoring sea conditions for patients when the within the context of Cooper at 19.

Alexander D. Macrae: In August and September, in close collaboration with principal investigators monitoring safe conditions for patients within the context of COVID-19, Sangamo dosed the first two patients in the Phase 1-2 STAR study evaluating ST920 gene therapy in Fabry disease. Dosing of the first cohort of this study is now complete, and enrollment is ongoing for cohort 2. We expect to share data on this study by the end of next year. During the quarter, we received additional regulatory approvals for the first in human phase 1-2 clinical study evaluating CAR-regulated T-cell, or CAR-Treg, candidate TX200 in kidney transplantation. We believe we are on track to initiate the study next year. Initiating this study may allow us to be the first company to explore the potential of CAR Treg cells in humans.

So I'm going to dose the first two patients in the phase one two star study.

Meeting or exceeding 20 gene therapy in fabry disease.

So it was it was the first cohort of the study is now complete.

Enrollment is ongoing for cohort two.

We expect to share diesel in this study by the end of next year.

During the quarter, we received additional regulatory approvals for the first in human Phase one two clinical study evaluating card break that she T cell or car T. Reg candidate, TX 200, and could peak transplantation.

We believe we are on track to initiate the study next year.

Initiating the study may allow us to be the first company to explore the potential of car T Reg cells in humans.

Alexander D. Macrae: We are hopeful that this will provide broader proof of concept for genetically engineered cell therapy using T-Rex. Beyond transplantation, we intend to further evaluate CAR Tregs, including zinc finger nucleus edited allogeneic Treg therapies in autoimmune diseases with high unmet medical need. Also this quarter, we completed our research activities associated with our ongoing Pfizer collaboration to develop gene regulation therapy using our zinc finger technology for the treatment of C9-ORF72 related ALS. In this program, our zinc finger proteins are designed to selectively target disease allele repeats, a remarkable demonstration of yet another way our versatile technology may be able to have a disease-modifying impact on challenging CNS diseases.

We are hopeful that this will provide broader proof of concept for genetically engineered cell therapy using T regs.

Beyond transplantation, we intend to protect all of your car T regs, including zinc finger nuclease attitude alert you need to break therapies in autoimmune diseases with Holly unmet medical need.

Also this quarter, we completed our research activities associated with the ongoing Pfizer collaboration to develop gene regulation therapy, using nursing finger technology for the treatment of obscene nine or 72 releases LLS.

This program or say finger proteins are just saying to selectively target disease I'll repeat a remarkable demonstration of yet. Another we are versatile technology may be able to have but to cease smelting thing impact when challenging CNS diseases.

Alexander D. Macrae: We recently earned a $5 million milestone payment from Pfizer associated with this program, which we expect to receive later this quarter. It is a further testament to our R&D momentum. We look forward to continuing to work closely with Pfizer to support their research and development efforts in this program. With that, I will turn the call over to our Chief Medical Officer, Bettina, who will provide additional details on our clinical accomplishments. Good afternoon.

We recently earned 5 million milestone payment from Pfizer associated with this program, which we expect to receive leads to this quarter.

It's a testament to our R&D momentum.

We look forward to continue to work closely with Pfizer to support their research and development in this program.

Because I would turn the call over to her Chief Medical Officer, Bettina, who will provide additional details on our clinical accomplishments.

Good afternoon.

Bettina M. Cockroft: As Sandy mentioned, our clinical operations have adapted to the challenges of the evolving COVID-19 pandemic, and we are pleased with our progress in executing on our partnered and wholly-owned program. Pfizer shows the first patient in the affine study of Giroctococcine Fetal Parvovec or SB525, our first acid in a registrational trial. AFINE is a global phase 3, open-label, multi-center, single-arm study evaluating the efficacy and safety of SB525 in patients with moderately severe to severe hemophilia A. The primary endpoint is annualized bleeding rate, or ABR, through 12 months following treatment. This will be compared to ABR while on Factor VIII replacement therapy collected in the Phase III lead-in study, which will provide a baseline for Phase III study participants. The secondary endpoints include factor VIII activity level after the onset of steady state over 12 months.

As somebody mentioned, our clinical operations have adapted to the challenges of the evolving COVID-19 pandemic and we're pleased with our progress in executing on a pocket and wholly owned programs.

Pfizer dosed the first patient in the Fine study drug took a gene so part of it or SP five to five well first I sit in a registrational trial.

A fine as a global phase three open label multi center single arm study evaluating the efficacy and safety of SP five to five in patients with moderately severe to severe hemophilia a.

The primary endpoint is on your life right, beating rights or IDR through 12 months following treatment.

This will be compared to a b R. Well in fact, the eight replacement therapy collected in the phase three data in study, which will provide a baseline for phase three study participants.

The secondary endpoints include factor eight activity level after the onset of steady state over 12 months.

Bettina M. Cockroft: Participants will be analysed throughout the five-year study period following the single infusion to further assess durability of efficacy and safety. Pfizer shared updated Phase 1-2 data at a Pfizer investor event in September, which demonstrated that SB 525 was generally well-tolerated. Each of the five patients in the Huidong cohort sustained a clinically meaningful level of factor VIII activity without bleeds or the need for prophylactic factor up to 85 weeks for the longest treated patient.

Participants will be analyzed throughout the five year study period following a single infusion to further assess your.

The rapidity of efficacy and safety.

Five to shed updated phase one two data out of Pfizer investor event in September which demonstrated that SB five to five was generally well tolerated.

Each of the five patients in the high dose cohorts sustained a clinically meaningful and that will affect freight activity without leads or the need for prophylactic fucked up to 85 weeks with the longest treated patients.

Bettina M. Cockroft: Both companies are encouraged by these results and plan to present further follow-up data from the ALTAR study in the next few months when all five patients in the 3E13 vector genomes per kilogram dose cohort have been followed for at least one year. We have dosed the first two patients comprising the first cohort in the Phase 1-2 STAR study evaluating ST920 Infabry disease. The goal of this gene therapy candidate is to provide a predictable and durable expression of the alpha-gal-A enzyme, which is deficient in Fabry disease due to mutations in the GLA gene, resulting in the accumulation of the substrate GB3 and its soluble derivative, lyso-GB3. This can cause challenging symptoms and morbidities, including impaired renal and cardiac function, pain, and gastrointestinal symptoms.

Both companies are encouraged by these results and plan to present for the follow up data from the Alta study in the next few months when all five patients in the 313 vector genomes per kilogram dose cohort has been followed for at least one year.

We have dosed the first two patients comprising the first cohort in the phase one to start study evaluating a few 920 in fabry disease.

The goal of this gene therapy candidate is to provide a predictable durable expression will be also Galway and sign which is deficient fabry disease due to mutations in the G.L.A. gene, resulting in the accumulation of the substrates gbpthree and its soluble derivative likes of Gbpthree.

This concludes challenging symptoms of morbidities, including impaired renal and cardiac function pain and gastrointestinal symptoms.

The Star trial is a multicenter open label dose ranging study evaluating the safety and Tolerability of us to 920 in classical fabry patients 18 years and older.

Bettina M. Cockroft: The STAR trial is a multi-center, open-label, dose-ranging study evaluating the safety and tolerability of ST920 in classical Fabry patients 18 years and older. Study participants will receive a single intravenous infusion of ST920, followed by one year of observation and monitoring of clinical endpoints, such as alpha-GalA activity and assessment of GB3 and lyso-GB3 levels. A long-term follow-up study will allow patients to be monitored for an additional four years. Enrollment for the second cohort is ongoing. We expect that data will be shared toward the end of 2021 after we have identified a dose for cohort expansion. We believe that ST920 offers a potentially differentiated treatment for Fabry disease, with the potential to deliver efficacy with preserved renal function and reduced cardiac morbidity and neuropathy. Preclinical studies evaluating ST920 demonstrated strong expression of alpha-GalA and GB3 substrate reduction across tissue types.

Study participants will receive a single intravenous infusion of a few nice 20, followed by one year of observation and monitoring of clinical endpoints, such as self ugly activity and assessment of Gbpthree and likes that you'd be three levels.

A long term follow up study will allow patients to be monitored for an additional four years.

Enrollment for the second cohort is ongoing.

We expect that they will be shed toward the end of 2021. After we have identified a dose cohort four cohort expansion.

We believe that I've seen I'm 20 offers a potentially differentiated treatment for fabry disease.

With the potential to deliver efficacy with preserved renal function and reduce morbidity in Europe at the.

Preclinical studies evaluating US Tonight, 20 demonstrated strong expression of alpha Galle and Gbpthree substrate reduction the trough tissue types.

That's delivered direct a gene therapy as senile 20 is delivered by a onetime intravenous infusion. It does not require any preconditioning regimen for patients.

We're also working closely with our oncology collaborator tight if you look at the company as it advances kites. They were three seven an allogeneic anti Cdnineteen car T therapy into a clinical trial.

Tight expects to submit an investigational new drug application by the end of 2020 and to initiate a clinical trial evaluating type 037 in Twentytwenty one.

Bettina M. Cockroft: As a liver-directed gene therapy, SC920 is delivered by a one-time intravenous infusion that does not require any preconditioning regimen for patients. We are also working closely with our oncology collaborator, KITE, a Gilead company, as it advances KITE037, an allogeneic anti-CD19 CAR-T therapy, into a clinical trial. TITE expects to submit an investigational new drug application by the end of 2020 and to initiate a clinical trial evaluating TITE-037 in 2021. Throughout the third quarter, we continued to receive additional regulatory approvals that support the Phase I-II Steadfast Clinical Study evaluating the first-in-human CAR Treg cell therapy Tx200 in HLA-A2 mismatched renal transplantation. We expect to initiate the study next year. The goal of this study is the prevention of transplant rejection through the engineering of Tregs to express an HLA-A2 chimeric antigen receptor, or CAR, allowing them to localize to the renal graft and activate upon recognition of the HLA-A2 antigen. CAR Tregs may prevent immune-mediated rejection through the inhibition and modulation of inflammatory immune cells and the release of anti-inflammatory cytokines to induce a tolerogenic environment within the graft.

Throughout the third quarter, we have continued to receive additional regulatory approvals that support the phase one two steadfast clinical study evaluating the first in human car T. Reg cells therapy, PX 200 in H.L.A. to mismatched renal transplant patients.

We expect to initiate the study next year.

The goal for the study is the prevention of transplant rejection through the engineering of T. Regs to express an h. delay to kind of metric antigen receptor or car, allowing them to localized to the renal graft and activate upon recognition to be actually a two antigen.

The car T regs may prevent immune mediated rejection through the inhibition and modulation of inflammatory immune cells and the release of anti inflammatory site took time to induce a toddler agenda environment within the crop.

Preclinical data supporting the steadfast study presented last month showed that the PX 208, Chile, a two car T regs sufficiently prevent rejection in both graft versus host disease and skin transplantation model.

They were also shown to be safe and well tolerated in our in vivo studies.

Similar to other genetically engineered cell therapy approaches patients will undergo a look apheresis procedure from which the T. Reg cells will be isolated and engineered the cryopreserved.

Bettina M. Cockroft: Preclinical data supporting the STEDFAST study presented last month showed that the PX200 HLA-A2 CAR-T regs efficiently prevented rejection in both graft-versus-host disease and skin transplantation models. They were also shown to be safe and well tolerated in our in vivo studies. Similar to other genetically-engineered cell therapy approaches, patients will undergo a leukophoresis procedure from which their Treg cells will be isolated and engineered, then cryopreserved. The HLA-A2 negative patient will subsequently undergo transplantation surgery and following a recovery period will receive their personalized TX200 drug candidate.

The h. delay to negative patients will subsequently undergo transplant patient surgery and following a recovery period will receive that personalized PX 200 drug candidate.

As a result of this detailed process, we expect those things patients will occur several months after their enrollment.

Recent publications show that the regulatory cell therapy space is gaining momentum and excitement in the scientific community.

In particular, the one study a large international technical study gathering seven investigator led trials across five countries showed that immune regulatory cell therapy as a whole was safe and that immune cell therapy is a potentially useful therapeutic approach in renal transplant recipients, allowing immune.

Cell composition restoration to normal healthy level and minimize section of the burden of general immune suppression.

This is very promising and supports our plan to evaluate car T regs in renal transplant patient populations.

Bettina M. Cockroft: As a result of this detailed process, we expect dosing of patients will occur several months after their enrolment. Recent publications show that the regulatory cell therapy space is gaining momentum and excitement in the scientific community. In particular, the one study, a large international clinical study gathering seven investigator-led trials across five countries, showed that immune regulatory cell therapies as a whole were safe and that immune cell therapy is a potentially useful therapeutic approach in renal transplant recipients, allowing immune cell composition restoration to normal healthy levels and minimization of the burden of general immunosuppression.

I will now turn the call over to some for an overview of the financial results. Some.

Thank you Bettina and good afternoon, everyone.

We used to share our financial results for the third quarter of 2020.

We reported a net loss of $1.6 million or one cents per share compared to a net loss of $27.3 million or 24 cents per share for the same period in 2019.

Total revenues were $57.8 million compared to $22 million for the same period in 2019.

The increase was primarily attributable to a 30 million dollar milestone achieved for SP five to five our hemophilia a candidate partnered with Pfizer and a $5 million milestone achieved for our Cnine or 72 collaboration with Pfizer.

Bettina M. Cockroft: This is very promising and supports our plan to evaluate CAR Tregs in renal transplantation populations. I will now turn the call over to Sung for an overview of the financial results.

Turning to expenses.

Non-GAAP operating expenses, which excludes stock based compensation expense were $54.8 million compared to $46.5 million for the same period in 2019.

Sung Lee: Thank you, Bettina, and good afternoon, everyone. We're pleased to share our financial results for the third quarter of 2020. We reported a net loss of $1.6 million, or $0.01 per share, compared to a net loss of $27.3 million, or $0.24 per share, for the same period in 2019. Total revenues were $57.8 million compared to $22 million for the same period in 2019. The increase was primarily attributable to a $30 million milestone achieved for SV525, our hemophilia A candidate partnered with Pfizer, and a $5 million milestone achieved for our C9ORF72 collaboration with Pfizer. Turning to expenses, non-GAAP operating expenses, which exclude stock-based compensation expense, were $54.8 million compared to $46.5 million for the same period in 2019.

The increase in operating expense reflects our head count growth and facilities expansion to support the advancement of our therapeutic pipeline and manufacturing capability.

These increases were partially offset by a decrease in clinical and manufacturing supply expenses.

Moving to the balance sheet.

We ended the quarter with $695 million in cash cash equivalents and marketable securities.

This balance includes a $75 million upfront license fee payment received from Novartis.

Additionally, in the current quarter, we expect to receive the $35 million milestone payments from Pfizer mentioned earlier.

We believe our balance sheet remains strong and will allow us to reach several important R&D milestones, including the potential filing of the BLE for SP five to five for hemophilia.

Turning to 2020 for your guidance.

We are updating our financial guidance for non-GAAP operating expenses, which exclude estimated stock compensation expense of $25 million from an estimated range of 210 million to $225 million to now be in the estimated range of 210 million to $220 million.

Sung Lee: The increase in operating expense reflects headcount growth and facilities expansion to support the advancement of our therapeutic pipeline and manufacturing capabilities. These increases were partially offset by a decrease in clinical and manufacturing supply expenses. Moving to the balance sheet, we ended the quarter with $695 million in cash, cash equivalents, and marketable securities.

I'll now turn it back to Sandy for closing remarks.

Thank you Sir.

We are focused on clinical execution and building momentum as.

As we adapt to the conditions of COVID-19, and head towards the end of the year.

We are pleased with our progress in clinical operations and with our partner programs.

A strong balance sheet enables us to advance our R&D pipeline.

We believe these accomplishments are could cycle in a strong position to achieve several important milestones and catalysts heading into next year.

Sung Lee: This balance includes the $75 million upfront license fee payment received from Novartis. Additionally, in the current quarter, we expect to receive the $35 million in milestone payments from Pfizer mentioned earlier. We believe our balance sheet remains strong and will allow us to reach several important R&D milestones, including the potential filing of the BLA for SV525 for hemophilia A. Turning to the 2020 full year guide. We are updating our financial guidance for non-GAAP operating expenses, which excludes estimated stock compensation expense of $25 million, from an estimated range of $210 million to $225 million to now be in the estimated range of $210 million to $220 million.

We believe we remain on track for a manufacturing facility in Brisbane to feel pretty sure at the end of this year and for cell therapy facilities, and brisman unfold to be operational by year end Twentytwenty one.

We anticipate continued improvement in Pfizer's piece three of Fame study with a pivotal data readout expected by Pfizer in Twentytwenty two Noksix state one year into your phase one two data presentations over the next year and Uh Huh.

We expect continued enrollment in the phase one to start study and the D to read data breach out towards the end of 20 to 21.

Sanofi has guided the first data readout from the phase one two sickle cell disease studies expected next year.

We anticipate presenting full look as T 400, pizza Telecity too at the same time.

And lastly, we expect the clinical trial initiation. So the phase one two first in human car T. Reg steadfast study will occur in 22 into one.

Alexander D. Macrae: I'll now turn it back to Sandy for closing remarks. Thank you so much. We're focused on clinical execution and building momentum as we adapt to the conditions of COVID-19 and head towards the end of the year. We're pleased with our progress in clinical operations and with our partner programs. Our strong balance sheet enables us to advance our R&D pipeline. We believe these accomplishments have put Sangamo in a strong position to achieve several important milestones and catalysts heading into next year.

And kite expects so that study is dilute unique anti Cdnineteen car T product candidate count for the second well.

We'll also commence and Twentytwenty one.

We look forward to delivering on these milestones in the coming year.

Operator, please open the line for questions.

Certainly.

Ask a question you will need to press star one on your telephone to withdraw your question press the pound key.

Please stand by while we compile the Q1 a roster.

And our first question comes from the line of Jeff meat.

With bank of America.

Hey, guys. Thanks for the question and congrats on all the pipeline progress.

Alexander D. Macrae: We believe we remain on track for our AAV manufacturing facility in Brisbane to be operational at the end of this year and for our cell therapy facilities in Brisbane and Vauban to be operational by year-end 2021. We anticipate continued enrollment in Pfizer's Phase 3 affine study with a pivotal data readout expected by Pfizer in 2022 and also expect one-year and two-year Phase 1-2 data presentations over the next year and a half We expect continued enrollment in the Phase 1-2 STAR study and a data readout towards the end of 2021. Sanofi has indicated that the first data readout from the phase 1-2 sickle cell disease study is expected next year.

I had a couple the first one is on the hemophilia study just with the 12 month Oh.

Annualized bleed rate and point I wanted to kind of get your feedback.

On on what FDA is looking for just relative to you know the feedback that Biomarin received and that's obviously a subject of a lot of investor conversations of late and then the second question is just a broader one on the strategy for the car T. Regs just wanted to maybe give us a little bit more color.

Text for you know, how you see that differentiating and maybe what successes you see what the best probability of success you see it in for example, solid tumors versus the liquid English. Thank you.

Thank you for your questions.

Alexander D. Macrae: We anticipate presenting follow-up ST400 beta thalassemia data at the same time. And lastly, we expect the clinical trial initiations of the Phase 1-2 first-in-human CAR Tregs FETPAS study will occur in 2021, and KITE expects that its study of allogeneic anti-CD19 CAR-T product candidate KITE-037 will also commence in 2021. We look forward to delivering on these milestones in the coming year. Operator, please open the line for questions. Certainly. To ask a question, you will need to press star 1 on your telephone.

On the first one I wrote in hemophilia, a we are limited in what we can see because the system and Pfizer sounds we are so pleased with their progress into phase three with their enthusiasm for the program.

The old way of content in their organization right up to their seat you haven't valuable they see this so for them.

Did.

You can be assured that they will be having regular conversations with the regulatory authorities and I'm certain that Pfizer will know how to navigate.

Landscape.

Regardless of T regs I'm going to pass over to Jason It's a real expert in this area and Jason can you talk to us about how do you see our two rig structure.

Thanks, Andy and.

Thanks for the question.

[music].

So.

First I'll start off by.

By pointing out that our programs and regulatory T cells engineered regulatory T cells are directed.

Operator: To withdraw your question, press the pound key. Please stand by while we compile the Q&A room. And our first question comes from the line of Jeff Meacham with Bank of America. Hey, guys. Thanks for the question, and congrats on all the pipeline progress. I had a couple.

In the auto immune and inflammatory.

In the space these are drugs for cancer.

So we're really excited about our engineered regulatory T cell platform and the programs that we're bringing forward.

Alexander D. Macrae: The first one is on the hemophilia A study. Just with the 12-month annualized bleed rate endpoint, I wanted to kind of get your feedback on what FDA is looking for just relative to, you know, the feedback that Biomarin received. I know that's obviously the subject of a lot of investor conversations of late.

We're excited about the progress and regulatory approvals that we read receipts.

Receipt so or.

About our first in human car T. Reg study TX 200.

We're leaders in this field and you.

You know, we are developing and refining our understanding of car T T Reg biology, and rapidly advancing our ability to engineer and manufacture that excel.

Alexander D. Macrae: And then the second question is just a broader one on the strategy for the CAR Tregs. Just wanted to maybe give us a little bit more context for, you know, how you see that differentiating and maybe what successes you see, what the best probability of success you see in, for example, solid tumors versus the liquid tumor study. Thank you. Thank you for your questions. On the first one around Haemophilia A, we are limited in what we can say because this is now in Pfizer's hands. But we are so pleased with their progress into Phase 3, with their enthusiasm for the program, and that all the way up and down their organization, right up to their CEO, how valuable they see this asset for them. You can be assured that they will be having regular conversations with the regulatory authorities, and I'm certain that Pfizer will know how to navigate that landscape. As regards Tregs, I'm going to pass this over to Jason, who is a real expert in this area.

And.

Our PX 200 study will be the first in human testing of engineered regulatory T cells of car T. Reg.

And this will be further demonstration of our leadership.

The goal with that study is the prevention of renal transplant rejection in the setting of an image. The mismatch transplant. So this is a setting where an H.L.A. to negative patient will receive an eight a. to positive kidney transplant.

In our therapy is comprised of T. Regs that are engineered to recognize the eight two antigen through a kind America antigen receptor and that a car that will drive the accumulation and activation of T. Regs again, the real growth.

And so.

Suppress the rejection of the gratified the patient's immune system and.

What's important about that study is that as I mentioned this is going to be the first test of this therapeutic hypothesis around car T Reg and just.

The study will be important for us to understand the safety and efficacy in the therapeutic potential of car T. Reg and we'll be informing programs that we're.

Jason D. Fontenot: And Jason, can you talk to us about how you see our Treg strategy? Thanks, Sandy, and thanks for the question.

Actively pursuing.

In larger autoimmune and inflammatory indications such as multiple sclerosis in Crohns disease.

Okay, great. Thank you.

Jason D. Fontenot: First, I'll start off by pointing out that our programs in regulatory T-cells, engineered regulatory T-cells, are directed in the autoimmune and inflammatory disease space. These aren't drugs for cancer. So we're really excited about our engineered regulatory T-cell platform and the programs that we're bringing forward. We're excited about the progress in regulatory approvals that we've received so far and about our first in human CAR Treg study, TX200. We're leaders in this field and, you know, we are developing and refining our understanding of Treg biology and rapidly advancing our ability to engineer and manufacture the cell. Our TX200 study will be the first in-human test of engineered regulatory T-cells of core Tregs, and this will be a further demonstration of our leadership. The goal of that study is the prevention of renal transplant rejection in the setting of an MHC mismatched transplant.

Thank you.

Our next question comes from the line of Maury Raycroft with Jefferies.

Hi, everyone. Thanks for taking my questions I had one on Vicki Reich program as well so I guess for getting that getting that study started can you talk more about what factors have led to pushing the study start to 2021 is it due to kobe to or the autologous cells.

Manufacturing process or anything else you can comment on and can you talk more about what else needs to be completed before starting this study.

Morry. Thank you very much for your question. We are we're very pleased with the progress of the T. Reg for actually two mismatch, we're very pleased with the approvals from the regulatory authorities we.

Cool for two separate were group it is in.

It is impacting the hospitals, we've begun to do the laporte trees. The manufacturing so there's a general cooled within <unk>, but we are confident that we will be able to move forward with this program.

Jason D. Fontenot: So this is a setting where an HLA-A2 negative patient will receive an HLA-A2 positive T cell therapy, and our therapy is comprised of Tregs that are engineered to recognize the A2 antigen through a chimeric antigen receptor. And that CAR will drive the accumulation and activation of Tregs in the renal graft and suppress the rejection of the graft by the patients. And what's important about that study is that, as I mentioned, this is going to be the first test of this therapeutic hypothesis around CAR T-REX. And this study will be important for us to understand the safety and the efficacy and the therapeutic potential of CAR Tregs, and it will inform programs that we're actively pursuing in larger autoimmune and inflammatory indications. Chorosis.

Got it and from a manufacturing and process development standpoint is that all figured out or is there any other perspective, you can provide on that.

One of the reasons that we acquired T. Excel in 22 team was their understanding of how to look after two rigs which is different from people who come from T cells and so we're very pleased with the progress that we've made.

Got it Okay and then the other question I had was just.

On the pre on program that you guys have or would you sort of under the radar I'm. Just wondering if you can provide a status update on that program.

Any timeline.

Update on that program and maybe talk about the strategic importance of that one as well.

Jason can you maybe talk at the prelims in what we think of them.

Sure Sandy thank you.

Jason D. Fontenot: Okay, great. Thank you. Thank you. Our next question comes from the line of Maureen Raycroft with Jeffrey. Hi, everyone.

The pre on program is in preclinical development. So I think you know we'll be looking forward to sharing updates at the appropriate time I think that.

Bettina M. Cockroft: Thanks for taking my questions. I had one on the TREG program as well. So I guess for getting that study started, can you talk more about what factors have led to pushing the study start to 2021? Is it due to COVID or the autologous cell manufacturing process or anything else that you can comment on? And can you talk more about what else needs to be completed before starting the study?

There is a great opportunity there to to demonstrate the.

The power of our platform similar to the approaches that we're taking with our partners.

In CNS Biogen and Novartis. These are both of these partnerships are driven by.

By what our partners see in our platform and the pre owned program is yet. Another example of that and we'll be excited to.

Bettina M. Cockroft: Maury, thank you very much for your question. We are very pleased with the progress of the Treg for HLA-A2 mismatch. We're very pleased with the approvals from the regulatory authorities.

To talk about it as we move forward.

And we've done some of the initial work with the broad Institute in Boston.

They have a real depicts the group, we're working with <unk> deep expertise in this and so.

Alexander D. Macrae: We COVID is everywhere, COVID is in, impacting the hospitals we go to, the laboratories, the manufacturing, so there's a general Covid impact, but we are confident that we'll be able to move forward with this program. Got it. And from a manufacturing and process development standpoint, is that all figured out? Or is there any other perspective you can provide?

We have stopped biological expertise to much with our technological expertise.

Got it. Thank you for the perspective, thanks for taking the questions.

Thank you Laurie.

Thank you and our next question comes from the line of Jim Birchenough with Wells Fargo.

Hi, Thanks for taking my question. This is again on for Tim So perhaps a question on the T Reg program as well.

It or could you confirm whether this is a gene attitude product because I think its autologous. So you know that obviously they eat too is put in with a gene addition approach.

Alexander D. Macrae: One of the reasons that we acquired TXL in 2018 was their understanding of how to look after Tregs, which is different from how people look after T-cells, and so we're very pleased with the progress that they've made. Okay. And then the other question I had was just about the PREON program that you guys have, which is sort of under the radar. I'm just wondering if you can provide a status update on that program, any timeline update on that program, and then maybe talk about the strategic importance of that one or something. Jason, could you maybe talk about prions and what we think of them... Sure, Sandy, thank you.

If you can comment on whether its a.

Gene edited product and then for your future product to the ATL generic regulatory car T cells.

How do you see the issue of persistent.

I guess for this program tier two X 200, as well how do you see the issue of persistence.

And for.

For renal transplant would you require long term persistent.

And would you explore repeat dosing for your animal <unk> program.

So thank you for your question do they're very sensible scientific questions and wouldn't let me try and limit the path could we choose and we choose to start with a totally go source, who can understand the effectiveness of T regs and one of the advantages all from.

Jason D. Fontenot: You know, the pre-on program is in pre-clinical development, so I think, you know, we'll be looking forward to sharing updates at the appropriate time. I think that there is a great opportunity there to demonstrate the power of our platform, similar to the approaches that we're taking with our partners in CNS, both Biogen and Novartis. Both of these partnerships are driven. Bye!

It will transform too soon to transplanted kidney compete biopsies because it's it's implemented close to the surface. So we can look at things like persistence, which as you see is an important.

Jason D. Fontenot: by what our partners see in our platform, and the PRION program is yet another example of that, and we'll be talking about it as we move forward. And we've done some of the initial work with the Broad Institute in Boston. And they have a real deep expertise in this; the group we're working with has a real deep expertise in this. And so we have that biological expertise to match our technological expertise. Thank you for the perspective. Thanks for taking the time to answer the question. Thank you, Maury.

Feature that would be required if any good treatment.

We will gradually switch to allogeneic and and we can either develop alerts uniques a T regs BOLI editing done from a healthy 200 volunteers or by starting on peak season other forms of stem cells to.

Alexander D. Macrae: Thank you. And our next question comes from the line of Jim Birchenhau with Wells Fargo. Hi, thanks for taking the call.

To develop them all to be T. Rex. So this is really at the very cutting edge of regulatory cellular signs and we are lucky to have as many options as possible to take us into series.

Alexander D. Macrae: My question, this is Yanan speaking on behalf of Jim, so perhaps a question on the TREG program as well. Could you confirm whether this is a gene-additive product? I think it's autologous, so... Obviously, the HLA-A2 is put in with a gene addition approach, the Allergene Additive Product, and then for your future product, the Allogeneic. Regulatory CAR T-cell, And how do you see the issue? I guess, for this program, TX200.

Got it and a quick follow up in PX 200, it there a gene editing component.

T X two hundreds is a toll lucasfilm under for dosing.

Gene editing component, Jason I'm correct on that.

Yes, that's correct.

Yes 200.

Alexander D. Macrae: How do you see the issue of persistence? For renal transplantation, it requires a long-term commitment. And would you explore or repeat those?

We introduced the car with a lentivirus.

For that first part of that first program, but obviously you.

Alexander D. Macrae: So, thank you for your question. They're very sensible scientific questions, and let me try and lay out the path that we've chosen, which is to start with autologous so that we can understand the effectiveness of Tregs. And one of the advantages of renal transplantation is that the transplanted kidney can be biopsied because it's implanted close to the surface, so we can look at things like persistence, which, as you will see, is an important feature that would be We will gradually switch to allogeneic Tregs, and we can either develop allogeneic Tregs by editing them down from healthy donor volunteers or by studying iPSCs and other forms of stem cells to develop them up to be Tregs. So this is really at the very cutting edge of regulatory cellular science, and we are lucky to have as many options as possible to take us into those areas. I got it.

Our platform one of the one of the assets we have a thing about is our platforms ability to do.

No because engineering of the T. Rex for future future products.

And that was why the marriage of Sanquin to excel was sensible. They they brought the two may be experiencing we broke editing that they needed to to the platform forward.

Got it.

And then maybe a question on zinc finger transcription factors.

Just wondering about the ore rich or region of the crush script transcription factor or whether it is 40 schulman.

Or is there any synthetic component into the transcript transcription factors and how do you think how should we think about immunogenicity. Thanks.

Jason do you want to have a go ahead. Please.

Sure. So so the basic components.

Of the zinc finger transcription factors are all human.

Alexander D. Macrae: And a quick follow-up in PX200. If there are gene-added... TX200 is an autologous form and therefore doesn't have a gene editing component. Jason, you're correct on that.

Obviously in order to to direct those transcription factors to a desired sequence we have to design a synthetic protein that is specific for specific sequencing the genome.

And by nature of the fact that they are fully human we expect that the immunogenicity should should be inherently lower.

Alexander D. Macrae: Yes, that's correct. TX200, we introduced the car with a lentivirus for that first program, but obviously, one of the assets we have at Sangamo is our platform's ability to do genomics, And that was why the marriage of Sangamo and TXL was so sensible. They brought the T-Rev experience, and we brought the editing that they needed to take the platform forward. Then, maybe a question on zinc finger transgrips. I'm just wondering about the original... whether it is a phony or if you're some synthetic.

And we haven't made any observations to date to suggest that the immunogenicity could be a problem.

Great. Thank you so much for taking my question and congrats on the progress.

Thank you very much.

Thank you.

Next question comes from the line of Gena Wang with Barclays.

Thank you for taking my questions I ask me questions well sources regarding hemophilia eight I'm, just wondering I'm not saying I know this is already tech transfer king size, So I'm, giving the bombing experience won two data.

Good morning. Thanks.

In three D. that did not.

Uh huh.

Hey, quite different from the first one to show any thoughts you can share with us to be Guardian walk phase one two data and never gotten.

Jason D. Fontenot: How do you... What should we think about immunosuppression? Jason, do you want to have a go at that, please? Sure, so the base components of the zinc finger transcription factors are all human, obviously, in order to direct those transcription factors to a desired sequence, we have to design a synthetic approach. I'm not sure about a specific sequence in the genome. By nature of the fact that they are fully human, we expect that the immunogenesis... should be inherently lower.

Also at the C suite data in a one hypothesis otherwise the manufacturing costs and other things you know this manufacturing phase one two data from your side and now we will catch back to Bell Vice or any thoughts you can help us understand in terms of potential Uh huh.

Really came from phase one two data that you're facing and then the second question is that we got from Pfizer.

Upon their programs at your last album thing a protein transcription gene regulation pool.

Jason D. Fontenot: And we haven't made any observations to suggest that. Thank you so much for taking the time. Thank you very much. Thank you. And our next question comes from the line of Gina Wang with Barclays. Thank you for taking my questions. I have three questions.

So when he can share any color regarding efficiency Oh from this initial idea Oh, Oh study and the last question is regarding desktop program.

Alexander D. Macrae: Now, the first is regarding hemophilia A. I'm just wondering, Sandy, I know this is already a tech transfer to Pfizer. Given the Balmering experience, Phase I-II data, you know, did not, all Phase III data did not have much difference from the Phase I-II data. So, you know, any thoughts you can share with us regarding your Phase I-II data and, you know, also the Phase III data?

Already known second cohort on what would be their determination that you think it will reach the optimal Oh cool, but you can expand a cohort all the call I'd say biomarker data like a class molecular GBC as.

As a reference.

We'll show, 30% to 40%, although the production, mostly as a baseline yachtsman any thoughts you have regarding what is and what the all in order to determining the optimal dose.

Alexander D. Macrae: And since, you know, this manufacturing Phase I-II is from your side and now will transfer to Pfizer, any thoughts you can help us understand in terms of potential predictability from Phase I-II data to Phase III? And then the second question is regarding Pfizer's partner program, the ALS gene-single-protein transcription gene regulation program. So, wondering if you can share any color regarding the efficiency of this initial R&D. And the last question is regarding the Fabry program; you've already enrolled the second cohort, and what would be the determination that you think you'll reach the optimal cohort, that you can expand the cohort regarding, say, biomarker data, like a plasma, like for GV3, as a reference, Avro showed 30 to 40% further reduction versus the So, Jane, I'm afraid I may disappoint you in my answers.

Hi, Jane I'm afraid on the disappointing in my answers the feis or.

The phase two of the Ultra study on the progression of this with Pfizer is really in their hands snow to talk about on the no interest or the abstracts for Ash, where I know just a d. and so they will be talking about that.

Oh, gosh, and so I would guide you to two week for produce results the confirms ability between face to face we really we we mustn't see anything until we see the phase three results.

As regards to see North all I can tell you spice are very pleased with the product could be produced for them and they have transferred into their research organization and paid the necessary Mount Sterling.

As regards fabry I'm going to pass on to Bettina between or can you talk a good.

Foundry import we're looking for and when to put this study will measure.

Alexander D. Macrae: The Pfizer and... The Phase 2 of the ULTRA study and the progression of this with Pfizer are really in their hands now to talk about. And they announced, or the abstracts for ASH were announced today, and so they'll be talking about that at ASH, and so I would guide you to wait for those results. The convertibility between phase two and phase three really we mustn't say anything until we see the phase three results. As regards C9-ORF, all I can tell you is Pfizer is very pleased with the product that we produced for them, and they have transferred it into their research organization and paid the necessary milestone. As regards Fabry, I'm going to pass it on to Bettina.

Absolutely.

So yes.

Yes. Thank you for the question so.

I've mentioned earlier, we dosed the first cohort.

To patients.

Enrollment is ongoing.

And that's.

The study goal really is to provide a predictable and durable expression of the also got me and decide which is the enzyme deficient and topic disease due to limitations in the jail 18.

So we are going to be measuring this parameter. It's one of the more and more important parameters that you point out we're also looking at substrates.

Gbpthree on life of Gbpthree.

These are.

The basis of some of the challenges in terms of mobility.

Bettina M. Cockroft: Bettina, can you talk about Fabry and what we are looking for and what the study will measure? Absolutely. So yes, thank you for the question. So, as I've mentioned earlier, we dosed the first cohort of two patients, and enrollment is ongoing. And the study goal really is to provide a predictable and durable expression of the alpha-GalA enzyme, which is the enzyme deficient in Fabry disease due to mutations in the GLA gene. And so we are going to be measuring this parameter as one of the more important parameters. But as you point out, we're also looking at substrates, GB3 and lyso-GB3. These are the basis of some of the challenging symptoms and morbidity in patients with Fabry. So we're going to be monitoring all of this data along with other data.

In patients with Fabry.

So we're going to be monitoring or that they say to them with other data remember this is first and foremost the safety and Tolerability study to start off with and.

We have a safety monitoring committee that will be will be involved in the decisions as we move on to <unk>.

Escalate goes to the optimum dose I think it is is with the totality of the data and the data that we are monitoring from.

Other studies with other products of course well.

Well that we will be.

Making our ultimate decision on me does that we bring forward in our cone product expansion.

Okay. Thank you.

Thank you very much for your question.

Bettina M. Cockroft: And remember, this is first and foremost a safety and tolerability study to start off with, and we have a safety monitoring committee that will be involved in the decisions as we move on to escalate our dose to the optimal dose. I think it is with the totality of the data and the data that we are monitoring from other studies with other products, of course, as well, that we will be making our ultimate decisions on the dose that we bring forward in our cohort expansion. Okay, thank you.

Thank you.

And our next question comes from a lot of air Joseph with JP Morgan.

Hi, Thanks for taking the question, maybe just perhaps it's usually one I'm curious to get a sense of whether there are.

Well, how we should be thinking about the potential for.

Additional largely wholly owned programs coming forth the clinic over the next 12 to 18 months relative to.

I guess, what we've seen over the past year, a fair amount of leveraging up the platform through partnering activities. Thanks.

Thank you for your question, Eric Mark the students like one for you. Please.

Alexander D. Macrae: Thank you very much for your question. And our next question comes from the line of Eric Joseph with J.P. Morgan. Hi, thanks for taking the question. Maybe just perhaps, a strategic one.

Hi, Eric Thanks for the question. So if you take a look at the good deals that we've done including those most recently.

Mark McClung: I'm curious to get a sense of whether there are, well, how we should be thinking about the potential for additional largely wholly owned programs coming to the clinic over the next 12 to 18 months, relative to, I guess what we've seen over the past year, a fair amount of, you know, leveraging of the platform through partnering activities. Thanks. Thank you for your question, Eric. Mark, this feels like one for you, please. Hi Eric.

Really the driver of that is two fold in some cases they've come to us.

You know obviously in the Biogen case interest in in the a couple of the candidates that we had progressing towards.

The the eye and D.

But they came to us with a view to actually expanding there.

Number of CMS targets that we weren't otherwise considering so we looked at that as being really an extension of our or potential pipeline.

Mark McClung: Thanks for the question. You know, so if you take a look at the deals that we've done, including those most recently, the driver of that is twofold. And in some cases, they've come to us, You know, obviously in the collagen case interest in, in, a couple of the candidates that we had progressing towards, um, the IND. But they came to us with a view to actually expanding the number of CNS targets that we weren't otherwise considering. So we looked at that as being really an extension of our potential pipeline. The second way we sort of take a look at partnerships is whether they're bringing specific expertise and the resources necessary to accelerate the products, assuming they're successful in patients. And in that particular case, as well as the Novartis case, both of those really fit those types of criteria.

The second way, we sort of take a look at partnerships is whether they're bringing a specific expertise and the resources necessary to accelerate the products, assuming they're successful to patients and in that particular case as well as the Novartis case, both of those really fit those type of criteria.

We fully intend and we too to become a genomics medicine company and we are continually looking to dance wholly owned assets, which we would take into the clinic and I think a good example of that is is what you heard in terms of.

T X 200.

As well as the follow on programs that we're intending to take forward into the clinic for our car T Red programs.

Got it I guess in addition to she Reg are there certain disease states.

Mark McClung: We fully intend to become a genomics medicine company, and we are continually looking to advance wholly owned assets that we would take into the clinic. And I think a good example of that is what you heard in terms of TX200, as well as the follow-on programs that we're intending to take forward into the clinic for our CAR T-REC program. Got it. I guess in addition to Treg, are there certain disease states?

Or target that you have.

Actually walled off.

And our private prioritizing for internal development or not.

Weve not disclosed that I mean, we're we're we're taking a look at obviously, we've got interest in the autoimmune space as we talked about with her car T red programs and Jason touched on earlier naturally we're working heavily within the CNS area with our partners in Biogen and.

Mark McClung: or targets that you have essentially walled off and are prioritizing for internal development or... We've not disclosed that. I mean, we're taking a look at, obviously, we've got an interest in the autoimmune spaces, as we talked about with our CAR-T reg programs and Jason touched on earlier. You know, naturally, we're working heavily within the CNS area with our partners in Biogen and our partnership with Novartis. And so, you know, there may be targets that we choose in that particular space, but we've not made any decisions on that as yet. We don't believe you can really stick it in the therapy area and then go after it. We need to let the science drive us towards things that we believe are important to develop for patients. I got it.

Our partnership with Novartis and so you know there may be targets that we choose in that particular space, but we've not made any decisions on that as as yet.

We don't believe you can't really stick out Gary and then go after and we need to let the science drives towards things that we believe are important to develop for patients.

Got it that's helpful. Thanks for taking the question.

Sure.

Thank you and our next question comes from the line of re to Baral with Cowen.

Hi, guys. Thank you for taking the question of supply lottery to just two quick questions from me first on the Star Fabry study are you still seeing impacts from cobot on enrollment I know you can put it. The first cohort are all the sites up and running and then as a quick follow up regarding the Pfizer collaboration with Daniela.

And you know what are the next steps for the program specifically, what's the next opportunity for a milestone to reach is that.

Mark McClung: That's helpful. Thanks for taking the question. Sure. Pleasure. Thank you. And our next question comes from the line for Ritu Baral with Calis. Hi guys, thank you for taking the question. This is Lila on... Two quick questions from me.

On contingent on them initiating a clinical trial any color on that would be helpful. Thank you.

Thank you for your question, we haven't we or Pfizer haven't commented on the last program and when the milestones will come.

I would say it again.

Drew mystery Park central and the coal script.

It's a remarkable piece of science to be able to suppress the transcription one automobile and lead author and one on touched his wife Pfizer came to us and we have achieved the requested of us and it's moved forward to them. So let's hope because it's such a dreadful disease like school because to patients.

Alexander D. Macrae: First, on the Star of Fabry study, are you still seeing impacts from COVID on enrollment? I know you've completed the first cohort, but are all the sites up and running? And then, as a quick follow-up, regarding the Pfizer collaboration with the ALS program, what are the next steps for the program? Specifically, what's the next opportunity for a milestone to be reached?

In this quarter.

Alexander D. Macrae: contingent on them initiating a clinical trial, and you color this with when the milestones will come. I would say again, I would reiterate what I've said in the call script. It's a remarkable piece of science to be able to suppress the transcription of one allele and leave the other one untouched. That's why Pfizer came to us, and we have achieved what they requested of us, and it's moved forward to them. So let's hope, because it's such a dreadful disease, let's hope it gets to patients as soon as possible. For the Fabry disease, I'm going to refer you to Bettina, who will answer that.

For the stoppages, each I'm going to refer you to Bettina who answer that question.

Yes, and thank you for that question. So on the clinical operations side I have to say, we have an excellent clinical operations team working very hard on.

Maintaining the relationship with all the sites.

During the content make we've also been able to.

Initiate sites qualify other sites as we expand our footprint for the study.

Of course covert has had an impact.

Alexander D. Macrae: Yes, and thank you for that question. So on the clinical operations side, I have to say, we have an excellent clinical operations team working very hard and maintaining the relationship with all the sites. And during the pandemic, we've also been able to initiate sites, and qualify other sites as we expand our footprint for the study. Of course, COVID has had an impact worldwide, some regions more than others.

Right.

Some regions more than others. We are confident that we can keep going well following the enrollment and dosing of the first two patients to keep going with our in Rome, and despite the pandemic at this stage where.

The things Weve put in place from an operational perspective with home visits and live virtual assistance has really helped us make sure that.

Bettina M. Cockroft: We are confident that we can keep going. We're following the enrollment and dosing of the first two patients to keep going with our enrollment despite the pandemic at this stage, where the things we've put in place from an operational perspective with home visits and virtual assistance have really helped us make sure that we, together with the sites, manage to guide the patients through the enrollment and screening procedures to ultimately get us to dosing and then follow up. I'm very pleased with the way the clinical team has handled it. We feel we have a responsibility to do this well, to make sure that the patient comes first, whether they're being treated for COVID, in which case we should not be taking up doctors' time.

We together with the site manage to.

To guide the patients through the.

The enrollment and screening procedures to ultimately get to two dosing and then follow up.

Got it very pleased to comment on.

I'm very I'm very pleased with the way the clinic would you have navigated we feel we have a responsibility to do this well to make sure that the patient comes first whether they are being treated for cools. We can bridge because we should not be.

Taking a doctor's times, but we've been ready to do it as soon as the window was open and I think the Tina and her team Joel.

To go so.

Thank you for the color.

Thank you.

Alexander D. Macrae: But we were ready to dose as soon as the window was open, and I think Bettina and her team have done a remarkable job to get us there. Thank you. Our next question comes from the line of Nicole Germino with Truist. Good evening, everyone. Hi, thanks for taking my question. In February, given the competition in this space, given what is more specifically about Sangamo's AECAPCID or the promoter that makes it differentiated and better than your peers? And how have you made that determination?

Next question comes from the line of Nicole Gamino with true.

Good evening, everyone Hi.

Hi, Thanks for taking my question on February.

Given the competition in this space or what is it more specifically that sangamo and agents have fit or the motor that make it a differentiated better than your peers and how he made that determination and.

Good how does he and I 20 impact the renal and cardiac tissue.

So let me take that one we are encouraged by the data we've seen from 86 in hemophilia B.

Alexander D. Macrae: And then how does ST920 impact renal and cardiac tissue? So let me take that one. We are encouraged by the data we've seen from AV6 in hemophilia, but until we dose patients with Fabry disease, we really can't and shouldn't comment on what it's going to look like. The animal data looks very encouraging. We achieve supermaximal dosing and a supermaximal effect.

Buffy to reduce patients with fabry disease, we really.

Can't and shouldn't comment on what's it's going to look like the animal data looks very encouraging we as a C achieve supramax move dosing and supermax will affect.

In the animal dosing that we recently published we were able to show benefit to boost the heart and the kidney.

But.

I'm, just prudent and telling people to wait and see what the clinical results are we getting the shoe the clinical results towards the end just next year when they do.

Alexander D. Macrae: And in the animal doses that we recently published, we were able to show benefits for both the heart and the kidney. But I'm, I'm just prudent in telling people to wait and let's see what the clinical results are. We expect to show the clinical results towards the end of next year when the dose escalation phase is completed. Okay, great.

Those eggs escalation piece is completed.

Okay, great. Thank you so much.

[noise]. Thank you and our next question comes from the line of Evan way with Guggenheim Securities.

Hi, guys. Thanks for taking my question I had two one on M&A and other on the hour car T program on him anyway.

Alexander D. Macrae: Thank you so much. Thank you. And our next question comes from Evan Wang with Guggenheim Security. Hi guys, thanks for taking my question. I had two: one on HIEM-A and the other on the Al-Qariqi program.

What's the plan going forward to share data on the phase one two I know you'll have to update at ash, but are there any specific time points prior to a complete picture of data and will not require kind of all patients are required across that time point.

And on our car T program, yet we've seen some.

Alexander D. Macrae: On HIEM-A, what's the plan going forward to share data on the Phase I-II? I know you'll have the update at ASH, but are there any specific time points to provide a complete picture of the data, and will that require all patients to cross that time point? And on the AlloCard T program, you know, we've seen some data, early data from other Allo programs, from Allogene, CRISPR, you know, any thoughts on the initial data generated so far and any kind of learnings you can take, especially given some of the deaths we've seen in those trials? So let me do the HEMA and then pass this to Jason to comment on ALO. So I'm going to say again what I've been saying throughout: Pfizer is in control of the release of data on Haemophilia A.

Data early data from.

Other aloe programs from Allergan CRISPR no any thoughts on the initial data generated so far and any kind of learnings.

I can take especially given some of.

The death rate seen in those trials.

So let me do the human and cost us to Jason to comment on.

So the I'm going to say again quite consumed to right size or who are.

Are in control of the release of data on human recently the.

There is an abstract has been accepted for ash and people use it for.

Conference schedule in the future to continue.

Continue to demonstrate the benefit to patients cold reduce absent bleeding events and no requirement for factor Jason can you talk about the.

Alexander D. Macrae: There is an abstract that's been accepted for ASH, and they will use whatever conference schedule in the future to continue to demonstrate the benefit to patients of reduced absent bleeding events and no requirement for factor. Jason, can you talk about the... Allogeneic question, please? Sure, thank you, Sandy. So, I believe that you're referring to alloCART-T programs in oncology and that we have a partnership, as you know, with Kite Gilead to support their allogeneic CART-T programs. KITE has guided us to begin the studies on their allogeneic CD19 targeted CAR T therapy next year, and we believe that the data that we've seen so far from others are consistent with these therapies having real therapeutic potential. We're excited about it, and we're excited to see it.

Allogeneic question. Please.

Sure. Thank you Sandy.

So [noise].

I believe that you're referring to our car T programs in oncology.

And now that we have a partnership.

With tight gilliatt to support their allogeneic car T programs.

Tight has has guided that we're expecting to begin the studies on on their allogeneic Cdnineteen targeted car T therapy next year, and we believe that the data that we've seen so far from from others are consistent with.

With these with these therapies, having real therapeutic potential.

We're excited about it.

And we're excited to see.

Jason D. Fontenot: Okay, thank you. Thank you. I will now turn the call back over to Head of Corporate Communications, Aaron Feingold, for any further remarks. Thank you once again for joining us today and for your questions. We look forward to keeping you updated on our future developments. Ladies and gentlemen, this concludes today's conference call. Thank you for participating, and you may now disconnect.

Okay, I get the get the therapies to patients.

Okay. Thank you.

Thank you.

I will now turn the call back over to head of corporate Communications, Aaron Finegold for any further remarks.

Thank you once again for joining us today and for your questions. We look forward to keeping you updated on our teacher and about.

Ladies and gentlemen, this concludes today's conference call. Thank you for participating and you may now disconnect.

[music].