Q1 2021 Sangamo Therapeutics Inc Earnings Call

Please be advised that today's conference is being recorded if you require further assistance. Please press star zero and I would now like day on the conference over to your speaker today ear and fine gold head of corporate Communications. Please go ahead.

Operator: Today's conference is being recorded. If you're requiring any further assistance, please press star zero. I would now like to hand the conference over to your speaker today, Erin Feingold, head of corporate communications. 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, including Sandy Macrae, Chief Executive Officer. Mark McClung, Chief Business Officer, Jason Fontenot, Chief Scientific Officer, Rob Schott, Head of Development, 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 on the events and presentations page. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to advancing our programs, plans, and timelines for enrolling and conducting clinical trials, and presenting

Good afternoon, and thank you for joining us today with me. This afternoon on this call are simple numbers of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer, Mark Mcclung, Chief Business Officer, Jason talk No Chief Scientific Officer Raj Shah head of debt.

And Bettina Cockroft, Chief Medical Officer.

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 single most current expectation.

And that's include but are not limited to statements relating to advancing our programs and plans and timelines for enrolling and conducting clinical trials presenting clinical data.

Erin Feingold: resumption clinical data, and

Erin Feingold: and Opening New Manufacturing Facilities, our 2021 Financial Guidance, our expectations regarding our financial performance and sufficiency of our cash resources, and other statements that are not historical facts. However, actual results may differ materially from what we discussed today.

And opening new manufacturing facilities, our 2021 financial guidance, our expectations regarding our financial performance and sufficiency of our cash resources and other statements that are not historical facts.

Actual results may differ materially from what we discussed today and these statements are subject to certain risks and uncertainties uncertainties that are discussed in our filings with the S. E. C. Specifically our annual report on form 10 and change for the fiscal year ended December 31st 20 times the.

Erin Feingold: These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically our annual report on Form 10-K for the fiscal year ended December 31, 2020. The forward-looking statements dated today are made as of this date, and we undertake no duty to update such information except as required by law. On this call, we discuss our non-GAAP operating expenses.

The forward looking statements stated today are made as of course date, and we undertake no duty to update such information, except as required by law.

On this call we discussed our non-GAAP operating expenses and reconciliation of this measure to our GAAP operating expenses can be found in today's press release, which is available on our website.

Alexander D. Macrae: 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, Aaron, and good afternoon to everyone on the call. This quarter, we have continued our focus on advancing our LEAD programs through clinical execution, regulatory interactions, and collaboration with our partners and investigators. This morning, Pfizer's CEO announced on their earnings call that progress continues with their partner to haemophiliate a gene therapy product candidate.

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

Thank you Erin and good afternoon to everyone on the call.

This quarter, we have continued our focus on advancing our lead programs through clinical execution.

Regulatory interactions and collaboration with our partners and investigators.

This morning, Pfizer CEO and notes on their earnings call. The progress continues with their partner to hemophilia, a gene therapy product candidate <unk>.

Alexander D. Macrae: Highlighting that the lead-in study for Pfizer's Registrational Phase 3 Affine Clinical Trial is now fully enrolled, which could lead to a pivotal readout as early as 2022. Together with Sanofi, we announce that the EMA has granted orphan designation to BIBB003, now known as SAR 445136, our cell therapy product candidate for the treatment of sickle cell disease. The orphan designation was based on preliminary clinical observations submitted to the agency.

And the delete and Scotty per Pfizer's, Registrational phase III, a fine clinical.

Clinical trial at least I was truly a goat, which could lead to a pivotal readout as early as 2022.

With Sanofi, we announced so the economy has grown to orphan designation to VIP piece you received a decree.

No no one is S. T R 4451, and three six our cell therapy product candidate for the treatment of sickle cell disease.

The orphan designation was based on preliminary clinical observations submitted to the agency.

Alexander D. Macrae: We in Sanofi also received fast track designation from the SDA for this program. We're pleased with this progress and expect to present initial data at a medical meeting by the end of 2021. In March, we initiated our steadfast clinical study evaluating TX200, our wholly owned CAR Treg cell therapy product candidate for renal transplant rejection, which we believe will be the first in-human clinical study of a CAR Treg therapy. We were pleased to open clinical sites sooner than expected and hope to have our first patient enrolled by the end of the year.

We incentive fee also received fast track designation from the SD eight for this program.

We're pleased with this progress and expect to present initial data to Mexico Beach and by the end of 2021.

Okay.

In March we.

We initiated our steadfast clinical study evaluating TX 200, our wholly owned car T Reg cell therapy product candidate and renal transplant rejection.

Which we believe will be the first in human clinical study of a car T therapy.

We were pleased to open clinical sites sooner than expected and hope to have a first patient and go by the end of the year.

Alexander D. Macrae: We see this study as the first step in our R&D journey towards allogeneic cardiac therapies for autoimmune diseases, which we hope to develop as wholly owned product candidates in our pipeline. Our research engine has been highly productive this quarter. We continue to focus on our innovative areas, including CAR Tregs for autoimmune diseases and transcriptional regulation with zinc finger proteins for neurological diseases. The versatility of our ZincFringer technology to address challenging diseases of the central nervous system was highlighted this quarter with data publications and presentations from our Tau and Alpha-Nuclein-Synuclein programs.

We see this study is the first step and our R&D journey towards Allogeneic car T therapies for autoimmune diseases, which.

Which we hope to develop this wholly owned product candidates and our pipeline.

Our research engine has been highly productive this quarter, we continued to focus on our various including car T regs for auction and diseases and transcriptional regulation and.

Proteins for neurological diseases.

The first utility overseen printer technology to address challenging disease of the central nervous system was highlighted this quarter with data publications and presentations from our toe and often you clean signed nuclear and programs.

Yeah.

Alexander D. Macrae: Before I turn the call over to the team, I'd like to take a moment to acknowledge that this quarter marked one year of working in the challenging conditions of the pandemic. I continue to be so impressed by the Sangamo team's resilience and determination as our lab employees have adapted to distancing procedures and as our remote colleagues tune into Zoom calls from kitchens and bedrooms. I'm proud of the strong team we have in place to execute on the capitalist risk-rich year we have ahead of us. With that, I will turn the call over to Rob Schott, our Head of Development, who will provide additional details on our clinical accomplishments. Good afternoon,

Before I turn the call over to his team I'd like to take a moment to acknowledge that this quarter marked one year of working and the challenging conditions of the pandemic.

I continue to be so impressed by the Sangamo team's resilience and determination and started lap employees have adapted to distancing procedures and there's a remote colleagues tune into zoom calls from kitchens and bedrooms.

I'm proud of the strong team they've been pleased to execute on the catalyst risk rich here, we have ahead of us.

With that I will turn the call over to Rob Shaw to tell me to provide additional details on our clinical accomplishments rope.

Rob Schott: Our development organization remains focused on execution in the clinic, and we are pleased with this quarter's progress. As Sandy mentioned, enrollment in Pfizer's lead study in the Phase 3 affine trial evaluating our hemophilia A product candidate is complete. The purpose of this study is to collect a minimum of six months of prospective efficacy data of current Factor VIII prophylaxis replacement therapy in patients with hemophilia A to establish baseline characteristics prior to dosing with Gorodotok gene filoparvivec in the affine trial.

Good afternoon.

Our development organization remains focused on execution and the clinic and we are pleased with this quarter's progress.

And as Sandy mentioned enrolment and Pfizer's <unk> study and the phase III fine trial evaluating our hemophilia a product candidate is complete and the purpose of this study is to collect a minimum of six months of prospective efficacy data of current factor eight prophylaxis replacement therapy and patients with hemophilia a to establish baseline.

Characteristics prior to dosing with career took gene so sort of par vivek and you'll find trial Pfizer expects a pivotal data readout as early as 2020 two as well as a two year update from the phase one two off the study and the fourth quarter of this year.

Rob Schott: Pfizer expects a pivotal data readout as early as 2022, as well as a two-year update from the Phase I-II ALTA study in the fourth quarter of this year. Regarding our FIBRI program, we continue to advance the Phase I-II STAR study, and we have recently enrolled the fourth patient. We plan to present initial data from this study in the fourth quarter of this year. In March, the EMA granted orphan designation to BIVV003, now known as SAR445136, our sickle cell disease product candidate partnered with Sanofi.

Regarding our Fabry program, we continue to advance the phase one two star study and we have recently enrolled the fourth patient and we.

We plan to present initial data from this study and the fourth quarter of this year.

In March the EMA granted orphan designation to D. I V V 003, now known as S. A our 445136, our sickle cell disease product candidate partnered with Sanofi.

Rob Schott: This decision was based in part on early data from three treated patients that had 52 weeks, 13 weeks, and 29 days of follow-up. In a recently published minutes, the EMA's Committee for Orphan Medicinal Products determined that preliminary clinical observations, as well as the potential of long-term effects that may obviate the need for frequent treatment, suggest a clinically relevant advantage versus hydroxyurea. Last month, Sanofi announced that this program received fast-track designation from the FDA.

This decision was based in part on early data from three treated patients that at 52 weeks 13 weeks and 29 days of follow up respectively and.

And a recent we published minutes the Ema's committee for orphan medicinal products determined that preliminary clinical observations as well as the potential of long term effects that may obviate the need for frequent treatments suggest a clinically relevant advantage versus hydroxyurea.

Last month's Sanofi announced that this program received fast track designation from the FDA.

We find the progress on this program encouraging we and Sanofi expect to present the initial data from the precision one study at a medical meeting by the end of this year, which will reflect a more mature and more comprehensive dataset.

Rob Schott: We find the progress on this program encouraging. We in Sanofi expect to present the initial data from the PRECISEN1 study at a medical meeting by the end of this year, which will reflect a more mature and more comprehensive data set than the early data shared with the EMA. Regarding our oncology collaboration as part of their recent portfolio review,

And then the early data on shared with the EMA.

Regarding our oncology collaboration as part of their recent portfolio review kite made a decision not to submit the kite <unk> seven and I N D. At this time, we are working closely with quite to determine the path forward for kite 037.

Rob Schott: KITE made a decision not to submit the KITE 037 IND at this time. We are working closely with KITE to determine the path forward for KITE 037. Together, we believe that KITE's oncology expertise and development experience, paired with Sangamo's ZyncFinger platform and cell engineering capabilities, positions the collaboration well within the space.

Our collaboration and remains focused on developing best in class oncology engineered cell therapies together, we believe that creates oncology expertise and development experience paired with sangamo and zinc finger platform and cell engineering capabilities positions the collaborations well within the space.

Rob Schott: Lastly, we are very pleased to have initiated our Phase I-II Steadfast Study earlier than expected. This study is evaluating TX200, our autologous HLA-A2 CAR Treg cell therapy product candidate in patients receiving an HLA-A2 mismatched kidney from a living donor. Patient recruitment is now open at clinical sites in Belgium and the Netherlands. The primary objective is to assess the safety and tolerability of TX200. Secondary objectives include the incidence of biopsy-confirmed acute graft rejection, incidence of chronic graft rejection, and localization of TX200 CAR Treg cells in the transplanted kidney.

Lastly, we are very pleased to have initial initiated our phase one two steadfast study earlier than expected.

This study is evaluating TX 200, our autologous HLA <unk> car T. Reg cell therapy product candidate in patients receiving and H L. A a two mismatched kidney from a living donor and.

Patient recruitment is now open at clinical sites in Belgium, and the Netherlands. The primary objective is to assess the safety and Tolerability of TX 200 <unk>.

Secondary objectives include the incidence of biopsy confirmed acute graft rejection incidents of chronic graft rejection and localization of TX 200 car T Reg cells and the transplanted kidney. We also plan to evaluate the ability to reduce systemic immunosuppressive therapy, which can be associated with SAP.

Rob Schott: We also plan to evaluate the ability to reduce systemic immunosuppressive therapy, which can be associated with side effects such as an increased risk of infections or other serious conditions. We plan to enroll up to 15 patients in the group receiving TX200 and up to six patients in a control group. We plan to evaluate three doses with three patients in each dose cohort with the option for an expansion cohort with up to six additional patients. The study will be monitored by an independent safety monitoring committee, which will give its go-ahead before moving to the next dose cohort. Patients in the TX200 group will undergo

Effects, such as and increase risk of infections or other serious conditions.

We plan to enroll up to 15 patients and the group receiving TX 200.

And up to six patients and a control group, we plan to evaluate three doses with three patients and each dose cohort with the option for an expansion cohort with up to six additional patients.

The study will be monitored by an independent safety monitoring committee, which will give it a go ahead before moving to the next dose cohort.

Patients and the TX 200 group will undergo a leukapheresis to collect white blood cells prior to their transplant surgery, we will isolate a subset of T. Reg cells and modify them to add a comerica antigen receptor or car to allow specific recognition of the H O a eight to protein present on the cells of the <unk>.

Rob Schott: The HLA-A200 group will undergo leukophoresis to collect white blood cells prior to their transplant surgery. We will isolate a subset of Treg cells and modify them to add a chimeric antigen receptor, or CAR, to allow specific recognition of the HLA-A2 protein present on the cells of the donated kidney. After transplant surgery and a several-month recovery period, the patient will receive their one-time individualized TX200 infusion. We expect to enroll the first patient in this study by the end of this year and expect that dosing will occur several months thereafter.

And a kidney.

And after transplant surgery.

And a several month recovery period, the patient will receive their one time individualized TX 200 infusion we.

We expect to enroll the first patient in this study by the end of this year and expect that dosing will occur several months thereafter.

We believe that by directing the T Reg cells to the transplanted organ by way of the Comerica antigen receptor. TX 200 has the potential to mitigate graft rejection and reduce the need for systemic immunosuppression. We see this study is offering and opportunity to demonstrate important proof of concept car T Reg biology and humans.

Jason D. Fontenot: We believe that by directing Treg cells to the transplanted organ by way of the chimeric antigen receptor, TX200 has the potential to mitigate graft rejection and reduce the need for systemic immunosuppression. We see this study as offering an opportunity to demonstrate important proof-of-concept CAR-Treg biology in humans, as well as to better understand CAR-Treg process development. I will now turn the call over to our Chief Scientific Officer, Jason Fontenot, for additional details on these research programs.

And as well as to better understand car T Reg process development.

I will now turn the call over to our Chief Scientific Officer, Jason Fontana for additional details on these research programs Jason.

Thank you, Rob and good afternoon, everyone.

Day I'm delighted to give you an overview of a few of our exciting research program.

We can engineer, our zinc finger proteins on the ft precisely target essentially clean a DNA sequence from the genome.

Ben and methods to via P interactions with DNA, the optimized specificity and precision and we've developed methods to pair this targeting capability with a range of functional domains.

Jason D. Fontenot: Thank you, Rob, and good afternoon, everyone. Today, I'm delighted to give you an overview of a few of our exciting research programs. We can engineer our zinc finger proteins or ZFPs to precisely target essentially any DNA sequence in the gene. We have invented methods to tune VFP interactions with DNA to optimize specificity and precision. And we have developed methods to pair this targeting capability with a range of functional domains. These functional domains include transcriptional activators and requests that can modulate gene expression without altering the genetic code.

And as functional domains include transcriptional activators and request.

That can modulate and gene expression without altering the genetic code and new.

<unk> cases that can edit DNA sequencing and we are advancing via targeted based on editors and work harmony.

We can deploy these tools in vivo or ex vivo to create powerful therapies with the potential to treat significant disease.

As Andy touched on we are using our zinc finger protein transcription factors and develop promising therapies for diseases of the central nervous system and had some exciting preclinical data highlights this quarter.

First.

Data from our Tau program in collaboration with Biogen published in Science advances.

Jason D. Fontenot: Nucleases that can edit DNA sequences, and we are advancing ZFP-targeted base editors and recombinants. You can deploy these tools in vivo or ex vivo to create powerful therapies with the potential to treat serious diseases. As Sandy touched on, we are using our zinc-finger protein transcription factors to develop promising therapies for diseases of the central nervous system, and we had some exciting pre-clinical data highlights this quarter. Theta for Martel program, in collaboration with Biogen, published in Science Advanced, showed that TAO targeted Dinkfinger transcriptional repressors delivered by a single AAV administrator selectively reduced tau messenger RNA and protein by 50 to 80 percent out to 11 million, this without detectable off target.

The Tau targeted zinc finger transcription and Rhopressa is delivered by a single AAV administration selectively reduced messenger RNA and protein by $50 to 80 per cent out to 11 months this without detectable off target effect.

Second data from our Alpha nuclear program also in collaboration with Biogen presented at the Alzheimer's and Parkinson's disease, California showed that alpha to nuclear and targeted zinc finger transcription on rhopressa.

Significantly request human Alpha from nuclear and were well tolerated and vivo.

And finally data from our Alpha nuclear and program as well as our C&I and or 72 program, which was exclusively licensed to Pfizer had been selected for presentation at the American Society of gene and cell therapy annual meeting.

We look forward to keeping you updated on these and other preclinical programs as we advance.

As Rob mentioned, our steadfast car T. Reg clinical study opens for enrollment this quarter.

Jason D. Fontenot: Second, data from our Alpha-Nuclein Program, also in collaboration with Biogen, presented at the Alzheimer's and Parkinson's Disease Conference, showed that alpha-synuclein-targeted ring-finger transcriptional repressors could significantly repress human alpha-synuclein and were well-tolerated in patients. And finally, data from our Alpha-synuclein program as well as our C9-ORF72 program, which was exclusively licensed to Pfizer, have been selected for presentation at the American Society of Gene and Cell Therapy annual meeting.

I wanted to provide some additional color on our research strategy with regards to our car T portfolio.

Regulatory T cells called T regs have potent natural immunosuppressive property and.

Have the potential to be therapeutically hornets to treat diseases characterized by unwanted or excessive immune activity.

And on modified Polyclonal T Reg and mix of T regs, which overall do not target a particular edge and had been studied and autoimmune diseases have been shown to be safe. We are developing antigen targeted car T. Reg and believe that our approach may overcome the limitation polyclonal T. Reg by directing the T regs to the realm.

And on disease tissue, where they will be activated and expanded upon car body with the target antigen.

Jason D. Fontenot: We look forward to keeping you updated on these and other preclinical programs as we progress.

The antigen targeted by our car T. Reg can be linked to the disease ideology or simply be localized in the disease tissue.

Jason D. Fontenot: Technical Program, if we advance. As Rob mentioned, our steadfast CAR T-RAG clinical study opens for enrollment this fall. I want to provide some additional color on our research strategy with regard to our CAR T-RED portfolio. Regulatory T-cells, called Tregs, have potent natural immunosuppressive properties and have the potential to be therapeutically harnessed to treat diseases characterized by unwanted or excessive immune activity. Unmodified polyclonal Tregs, a mix of Tregs that overall do not target a particular antigen, have been studied in autoimmune diseases and have been shown to be safe.

For example, and our multiple sclerosis program.

Program and car T regs to target and Mylan Oligodendrocyte glycoprotein maag.

Mogg is express specifically and the central nervous system and similarly to a G. P. S. We will aim to drive our <unk> specific car T regs to expand at the site of inflammation, where they could suppress the pathogenic immune reaction responsible for them.

And our inflammatory bowel disease or IBD program.

We're targeting the receptor for the interleukin IL 23, our IL 23, R, which is known to be expressed and sizable ongoing inflammation and that is involved and IBD pathology.

Both programs were investigating multiple target answer.

We're using our zinc finger genome engineering platform to develop allogeneic T regs.

Edited T regs from healthy donors ex vivo to produce off the shelf car T regs out there we.

Jason D. Fontenot: We are developing antigen-targeted CAR Tregs and believe that our approach may overcome the limitations of polyclonal Tregs by directing the Tregs to the relevant disease tissue where they will be activated and expanded upon CAR binding with the target. The antigens targeted by our CAR Tregs can be linked to the disease etiology or simply be localized in the disease tissue. For example, in our Multiple Sclerosis Program, we are programming CAR-T regs to target myelin-oligodendrocyte glycoprotein, or MOG.

We are also investigating the potential use of induced pluripotent stem cells per Ips CS as a renewable source of large quantities of T regs, though.

We are making progress on these programs working first and developing efficient and proprietary car structure and.

And then on obtaining proof of concept preclinical data both in vitro and in vivo, we look forward to presenting our preclinical data in due course.

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

Thank you Jason and good afternoon, everyone is there and had mentioned previously our financial results for this quarter are available and the press release issued this afternoon, which can be found on our website.

Jason D. Fontenot: MOG is expressed specifically in the central nervous system, and similarly to a GPS, we will aim to drive our MOG-specific CAR Tregs to expand at this site of implementation, where they could suppress the pathogenic immune reaction responsible for it in our Inflammatory Bowel Disease, or IBD, program.

This quarter, we invested and the advancement of our clinical programs moving forward, our preclinical research pipeline and continuing to build our in house manufacturing capabilities.

We ended the quarter with approximately $630 million and cash cash equivalents and marketable securities.

We believe that our balance sheet remains strong and all.

And now us to reach several important R&D milestones.

And the potential submission of the BLA for our hemophilia a product candidate.

Mark McClung: We are targeting the receptor for the interleukin IL-23, or IL-23R, which is known to be expressed at sites of ongoing inflammation in the gut as involved in IBD pathology. Both programs are investigating multiple targets. We're using our ZincFinger genome engineering platform to develop allogeneic T-rays, edited Tregs from Healthy Donors Ex-Vivo to produce off-the-shelf CAR Tre We are also investigating the potential use of induced pluripotent stem cells, or iPSCs, as a renewable source of large quantities of Tregs.

Turning to 2021 full year guidance.

I'd like to reiterate the guidance, we provided on our prior call.

We continue to expect non-GAAP operating expenses, which excludes estimated noncash stock based compensation expense of approximately $30 million B and a range of approximately 255 billion to $275 million per the year.

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

Thank you Mark.

I'd like to conclude by saying that we're pleased with the progress this quarter across our development and research organizations and continue to believe we have the balance sheet strength to execute on our R&D milestones.

Mark McClung: We are making progress on these programs, working first on developing efficient and proprietary car structures and then on obtaining proof-of-concept preclinical data, both in vitro and in vivo. We look forward to presenting our preclinical data in due course. I will now turn the call over to Mark for an overview of the financial results. Thank you, Jason. And good afternoon, everyone.

Our manufacturing team continues to advance our in house cell therapy manufacturing facilities, which we expect to be operational by the end of the year.

We look forward to chemo students and catalyst by year end, including initial data readouts from sickle cell and February as.

As well as a two year update and hemophilia a.

Operator, please open the line for questions.

Thank you as a reminder to ask a question you will need to press star one on your telephone.

Mark McClung: As Aaron mentioned previously, our financial results for this quarter are available in the press release issued this afternoon, which can be found on our website. This quarter, we invested in the advancement of our clinical programs, moving forward our preclinical research pipeline, and continuing to build our in-house manufacturing capability. We ended the quarter with approximately $630 million in cash, cash equivalents, and marketable securities.

Withdraw your question press the pound key please standby, while we compile the Q&A roster.

Our first question comes from the line of Luca you think from RBC. Your line is now open.

Oh perfect. Thanks for taking the question. This is Lisa on for Lucas from RBC.

And just wanted to ask though we saw yesterday.

On that.

I will no longer be able to pursue and accelerated approval pathway for fabry disease that they may need to run a phase three trial head to head versus the standard of care and stabilize so and just wondering what your reaction was to that and is and how does that influence your development strategy going forward for you.

Mark McClung: We believe that our balance sheet remains strong and will allow us to reach several important R&D milestones, including the potential submission of the BLA for our hemophilia A product plan. Turning to 2021 full-year guidance, we would like to reiterate the guidance we provided in our prior call. We continue to expect non-GAAP operating expenses, which exclude estimated non-cash stock-based compensation expense of approximately $30 million, to be in the range of approximately $255 million to $275 million for the year.

Our gene therapy product. Thank you.

Thank you for your question.

Each one each company has their individual discussion with the agency.

You've been working on country for some time line.

Alexander D. Macrae: I'll now turn it back to Sandy for her closing remarks. Thank you, Mark. I'd like to conclude by saying that we're pleased with the progress this quarter across our development and research organizations and continue to believe we have the balance sheet strength to execute on our R&D milestone. Our manufacturing team continues to advance our in-house cell therapy manufacturing facilities, which we expect to be operational by the end of the year. We look forward to key milestones and catalysts by year-end, including initial data readouts on sickle cell and Fabry, as well as a two-year update on Haemophilia A.

What was your take on debt.

On insulin.

Yes, sure Sandy. Thank you well first of all what we are doing with currently focusing on pricing full force ahead on enrolling.

And when that phase one two study and as Rob.

Rob mentioned, we have dosed three patients and have enrolled four patients who meet.

And we'll hope to dose soon.

We are looking forward to sharing initial data readout in Q4.

Alexander D. Macrae: Operator, please open the line for questions. Thank you. As a reminder, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Please stand by while we compile the Q&A roster. Our first question comes from the line of Luca Issi from RBC. Your line is now open. Oh, perfect. Thanks for taking the question. This is Lisa. I'm for Luca from RBC.

Study design includes collecting biomarker data.

We've mentioned and the path as well, we will conduct kidney biopsies at the appropriate time.

The study is currently unaffected by the recent regulatory updates that youre referencing and for sure. We are engaging with the agency and at this point and sort of appreciate it.

Alexander D. Macrae: Just wanted to ask, so we saw yesterday that Avrobio will no longer be able to pursue an accelerated approval pathway for Fabry disease, but they may need to run a phase three trial head-to-head versus standard of care for Fabry.

It's too early to share and update on that from.

Thank you Bettina.

Great. Thanks for taking the question.

Thank you. Our next question comes from the line of Maury Raycroft from Jefferies. Your line is now open.

Operator: So, we're just wondering what your reaction was to that news and...

Hi, this is on.

And my recall for TX 200, and we notice.

Operator: And how does that influence your development strategy going forward for your gene therapy product? Thank you. Thank you for your questions. Each one, each company has its individual discussion with the agency. Bettina, you've been working on Fabry for some time now.

A bit earlier.

And press release.

And what work needs to be.

And before the patient enrollment and second half of 'twenty one.

Thank you for your question, Yes, we were pleased with the clinical Accretions group managing to initiate sites I think they've done a remarkable job and the time of COVID-19.

Bettina M. Cockroft: What did you think, what was your take on that announcement? Yes, sure, Sandy. Thank you. Well, first of all, what we are doing is currently focusing on pressing full court ahead with enrollment in the FABRI study, that Phase 1-2 STARS study. And as you've heard Rob mention, we have those three patients and have enrolled a fourth patient who we hope to enroll, and we hope to do so soon. We are looking forward to sharing initial data readout in Q4.

And no it's simply a matter of moving forward.

And.

And and enrolling patients and preparing for the dosing of the patient. This is the first time that we dosed a patient with a car T. Reg and so we're going to take care and be prudent making sure we have the right patient on the right.

Bettina M. Cockroft: Now, our study design includes collecting biomarker data, and as we've mentioned in the past as well, we will conduct kidney biopsies at the appropriate time. The study is currently unaffected by the recent regulatory updates that you're referencing, and for sure, we are engaging with the agency at this, but at this point, as you'll appreciate, it's too early to share an update on that front. Thank you, Betty.

And your factory and process to be able to drive forward and do this well and hopefully show that two rigs and potential is.

Matches, everyones excitement and remco, Syria to Louis to move into many autoimmune conditions.

Right.

Thank you. Our next question comes from the line of Jeff Meacham from Bank of America. Your line is now open.

Okay.

Hey, guys, it's Aspen on for.

Operator: Great. Thanks for taking the questions. Thank you. Our next question comes from the line of Maury Raycroft from Jefferies. Your line is now open. Hi, this is Kenny Chen on behalf of Maury Raycroft.

Yes, thanks for taking the questions.

Just two quick ones for US first of all Sandy I'd love to hear your thoughts on the current regulatory environment.

And specifically.

Respected seeber.

Operator: For TX200, we noticed the trial initiated a bit earlier compared to the 4Q press release. What were you saying? Thank you for your question. Yes, we were pleased with the clinical operations group managing to initiate sites. I think they've done a remarkable job during the time of COVID. And now it's simply a matter of moving forward and enrolling patients and preparing for the dosing of the patient. This is the first time that we've dosed a patient with a CAR-T reg, and so we're going to take care, be prudent, make sure we have the right patient and the right manufacturing process to be able to drive forward and do this well and hopefully show that CAR-T reg's potential matches everyone's excitement around this area to allow us to move into many autoimmune conditions.

Sort of a reduced communication and are you feeling any sort of shifting mentality from the agency.

And and respect.

And then secondly, just looking for a little bit more clarity on the on types decision not to submit the IND.

I wanted to see if you had any initial feedback and share with us. Thanks.

So let me let me take the more philosophical one and then I'll pass it on to Mark for the business discussion around guidance.

And so this week that feature marks given and awards for.

For his service to advancing medicines and it feels totally appropriate we have.

A great relationship with Seabourn, they are doing a difficult Joel and our flexible and thoughtful.

Operator: Thank you. Our next question comes from the line of Jeff Meacham from Bank of America. Your line is now open. Hey guys, it's Aspenon for Jeff.

I often get asked is has the regulatory environment got harder and be asking more and I would reflect the other way around.

Selling gene therapy with this one is this wonderful concept that will are so excited volume and no, but we're meeting as the reality of what it takes to health and medicine approved it's about ensuring and manufacturing is is good yourself process is good.

Operator: Thanks for taking the questions. Just two quick ones for us. First off, Sandy, I would love to hear your thoughts on the current regulatory environment, specifically with respect to CBER. Has there been any sort of reduced communication, or are you feeling any sort of shift in mentality from the agency in any respect? And secondly, I'm just looking for a little bit more clarity on the CAIC's decision not to submit the IND. I just wanted to see if you had any additional feedback you could share with us. Thanks.

Sciences' Good day clinical trial is good and it's not surprising that the.

<unk>.

Some of the excitement is no metering reality.

We take.

Take the stages extremely seriously sangamo, we invest greatly and CMC regulatory interactions and manufacturing and.

Alexander D. Macrae: So let me take the more philosophical one and then I'll pass it on to Mark for the business discussion around CHI. I saw this week that Peter Marks got given an award for his service to advancing medicines, and it feels totally appropriate. We have had a great relationship with CBER. They are doing a difficult job and are flexible and thoughtful. I often get asked, Has the regulatory environment got harder? Are they asking more? And I would reflect it the other way around.

And our.

Our.

Comfortable and confident that the agency hasnt changed its opinion, but on.

I'll pass on to Mark Cano for Kite Gilead, Mark do you want to talk about that.

Sure I mean.

Rob reiterated.

Kite made the decision not to submit to the $3 seven I and D. At this time and the team is going to look a little bit closer on to understand that potential path forward. This has been made and the context and the emerging.

Early data from.

Alexander D. Macrae: Cell and gene therapy is this wonderful concept that we all are so excited about. And now, what we're meeting is the reality of what it takes to have a medicine approved. It's about ensuring that your manufacturing is good, your cell process is good, your science is good, and the clinical trial is good. And it's not surprising that some of the excitement is now meeting reality, comfortable and confident that the agency hasn't changed its mind. But I'll pass on to Mark now for tight Gilead. Mark, do you want to talk about that?

From two competitors that were ahead of the <unk> hundred seven program, but also on the context of.

The date and it has on hand, and the startup product, which.

And the focus is really to drive something which.

Would be a best in class alternative and will allow for an expansion of the utilization of these products and so the goal is to continue to apply price expertise and the development of oncology paired with our zinc finger platform and cell engineering capabilities to really look.

Our opportunities to develop and advance best in class oncology and and engineered cell therapies.

Thanks, guys.

Thank you. Our next question comes from the line of Jan on <unk> from Wells Fargo Securities. Your line is now from.

Mark McClung: Sure. I mean, you know, as Rob reiterated, KITE made the decision not to submit the 037-IND at this time, and the team is going to look a little bit closer to understand that potential path forward. This has been made in the context of the emerging early data from two competitors that were ahead of the 037 program, but also in the context of, you know, the data that KITE has on hand with their Discarda product, for which the focus is really to drive something which would be a best-in-class alternative and would allow for an expansion of the utilization of these products.

Hi, Thanks for taking my questions I have a question regarding TX 200.

And I see that it's enrolling and.

And Belgium and Netherlands.

I'm wondering is there any difference and in terms of debt.

And the suppression and that is used for the renal transplant procedure any difference between Europe and the U S and if it's there.

There is some difference.

How would you also plan to open and IND and the U S tend to have a more relevant.

Treatment procedure for the U S a F.

Mark McClung: And so the goal is to continue to apply type expertise in the development of oncology paired with our zinc finger platform and cell engineering capabilities to really look for opportunities to develop and advance best-in-class oncology and engineered cell therapies. Thanks, guys. Thank you. Our next question comes from the line of Yanan Zhu from Wells Fargo Securities. Your line is now open. Hi, thanks for taking my questions. I have a question regarding T

Thanks.

Thank you for your question Russell interest in question, because renal transplant and how you treat it as is.

And as some.

Differs a little from country to country Bettina would you feel comfortable to talk about that.

Yeah, sure I mean, Sandy U U S debt.

Okay.

Differences from country to country.

And addressing your question on renal transplant.

Our.

Study as we've mentioned.

Recently initiated and we are enrolling in those two countries.

Operator: I see that it's enrolling in Belgium and the Netherlands. I'm wondering if there is any difference in terms of the immunosuppression that is used for the renal transplant procedure, any difference between Europe and the U.S.? And if there is some difference, how would you also plan to open an IND in the U.S. to have a more relevant treatment procedure for the U.S.

Mentioned, and Netherlands, and Belgium, we do intend to open study also in other countries.

Especially and other European countries. So this is something that.

We will hopefully be able to update and.

And the future with.

I might also add this is rob shot that Theres, often institution to institution differences in immunosuppressive therapies, and and you can even find differences within institutions between investigators.

Operator: application. Thanks.

Operator: Thank you for your question. That's an interesting question because renal transplantation and how you treat it differs a little from country to country. Patina, would you feel comfortable to talk about that?

And we rely on the investigator judgment with respect to immuno suppression and <unk>.

Bettina M. Cockroft: Yes, for sure. I mean, Sandy, you've said that there are differences from country to country in addressing immunosuppression and renal transplantation. We are, The study, as we've mentioned, has recently begun, and we are enrolling in these first two countries. You mentioned the Netherlands and Belgium. We do intend to open the study also in other countries, especially in other European countries. So this is something that we will hopefully be able to update you on in the future.

Therapy consequent or following the.

TX 200 therapy.

And.

Got it that's very helpful. And then maybe a follow up on the C&I or from 72 program could you talk about the potential advantages for it using the zinc finger protein transcription factor versus existing.

Gene therapy that delivers.

S. H RNA for example, and her knockdown of target gene and thank you.

Bettina M. Cockroft: I might also add, this is Rob Schott, that there are often institution-to-institution differences in immunosuppressive therapies, and you can even find differences within institutions between investigators. And we rely on the investigator judgment with respect to immunosuppression and therapy subsequent or following, and TX200 Therapy.

Sure would be happy to Jason can you help us with thoughtful.

Sure.

I think you know.

The key the key difference is really about about the platform technology about our platform and technology and our ability to.

To fine tune the interaction of our zinc finger proteins with.

Operator: Got it got that that's very helpful and then maybe a follow-up on the C9-ORF 72.

And the designated target so.

Operator: The C9ORF72 program, could you talk about the potential advantages of using the zinc finger protein transcription factor versus using a gene therapy that delivers SH RNA, for example, to knock down a target gene? Thank you. Sure, we'd be happy to. Jason, can you help us with that?

And so we have very far and control over that and we have the ability to identify and.

And you can think of proteins can accomplish what we set out to accomplish with the therapy and not have any off target and I think that there's a.

On a major difference between our ability to do that would be and clinker proteins and competitors.

And then beyond that from a clinical point of view.

Jason D. Fontenot: Sure. I think, you know, the key difference is really about the platform technology, about our platform technology, and our ability to... fine-tune the interaction of our zinc-finger proteins with... a designated target. So we have very fine control over that, and we have the ability to identify a zinc-synchroprotein that can accomplish what we set out to accomplish with the therapy and not have any off-target effects. And I think that there's a major difference between our ability to do that with zinc-synchroproteins and competitors.

On the R&D therapies seem to need to be gives and frequently and.

And we would talk that.

And that consumable expression of the transcription factor would give a long lasting effect it seems to do it and animals.

And until we get into humans, we won't know for sure, but the promised as gifts to patients although once and on therapy is really important.

These are dreadful diseases, and if we can address them with our transcription factor, we will fulfill our mission and promise.

Jason D. Fontenot: And then, beyond that, from a clinical point of view, the RNA therapies seem to need to be given frequently, and we would hope that epizomal expression of a transcription factor would give a long-lasting effect, as it seems to do in animals.

Great. Thanks for the color.

Okay.

Thank you. Our next question comes from the line of Gena Wang from Barclays. Your line is now open.

Jason D. Fontenot: Until we get into humans, we won't know for sure. But the promise this gives to patients of a once-and-done therapy is really important, these are dreadful diseases, and if we can address them with our transcription factor, we will fulfill our mission and promise. Great. Thanks for the cover. Thank you. Our next question comes from the line of Gina Wang from Barclays. Your line is now open.

Hi, This is Bob net on for Jade. Thanks for taking our questions. So I hired a fabry question.

Since looks like GBP, three reduction and kidney biopsies could serve as a surrogate endpoint is it safe to assume like you are predominantly focused on recruiting.

Patients of it could meet effect and.

And do you think that FDA would require longer term follow up commodity that egfr them to grant full approval based on the on the Fabulous time update thanks.

Operator: Hi, this is Swapanarayan Paljina. Thanks for taking our questions. So I had a Fabriry question. Since it looks like GB3 reduction in kidney biopsies could serve as a surrogate endpoint, is it safe to assume that you are predominantly focused on recruiting patients with kidney defects? And do you think that FDA would require longer-term follow-up to monitor EGFR to grant full approval based on the Fabrizyme update? Thank you.

Thank you for your question. The agency is doing it started best in my mind should make the pathway forward for fabry clear.

And on track to go.

What they have said is GBP three reduction and the kidney with the commitment to the long term Egfr benefit is pathway to.

To approval.

And therefore.

Alexander D. Macrae: Thank you for your question. The agency is doing its very best, in my mind, to make the pathway forward for Fabry clear and tractable. What they have said is that GB3 reduction in the kidney with the commitment to have a long-term EGFR benefit is a pathway to approval. And therefore, I think that

I think that on.

Understanding the effect on the kidney is important.

We are recruiting patients are on.

And on.

Enzyme replacement therapy or.

Our naive on Earth.

<unk> and the current study.

We are looking to show a biochemical effect and then we will move towards doing kidney biopsies.

Alexander D. Macrae: Understanding the effect on the kidney is important. Now, we are recruiting patients that are on enzyme replacement therapy, are naive, and are pseudo-naive in the current study. We are looking to show a biochemical effect, and then we will move towards doing kidney biopsies. So, simply, the patient population is wide-ranging, from patients that have already had renal damage to those that are undamaged, and what my reading of the literature tells me is that this is an important consequence to the patients and that patients show renal damage from very early on. So in amongst that population that we're already recruiting, there will be patients with renal consequences. But let's wait, let's see the results at the end of the year, and let's work out a sensible path forward.

So.

I think.

Simply.

The.

The patient population is as wide ranging from patients have already hunt.

And renal.

<unk> to those the R. R.

Undamaged.

And my.

My reading of the literature tells me is the.

This is an important consequences to the patients and the patient Shaw renal damage from very early on so and amongst the population that we're already recruiting and the whoopee patients with renal consequences.

But let's wait let's see the results at the end just easier and much work of essential path forward.

Operator: Thanks for taking our questions.

Got it thanks for taking my questions.

Operator: Thank you. Our next question comes from the line of Ritu Baral from Cowen. Your line is now open.

Thank you. Our next question comes from the line of Ritu <unk> from Cowen. Your line is now open.

Operator: Good afternoon, guys. Thanks for taking the questions. It's actually me. I wanted to ask you about the rationale for picking HLA-A2. On slide 11 of your corporate deck, you note that 21 to 26% of the transplanted renally transplanted organs are HLA-A2 mismatched. First of all, I wanted to make sure that the 21 to 26 were renal, were kidneys and not just organs in general.

Good afternoon guys.

Thanks for taking the questions.

Actually Luke.

I'm wondering about the rationale for picking HLA a two on slide 11.

And.

Corporate debt.

You'll note that 21 to 26 per cent of the transplanted runaway trim granted organs are HLA too.

On to mismatch first of all I wanted to make sure that that 21 26 was Reno was coming from not just organ from general and second.

Alexander D. Macrae: And second, in your comments today, between MS and IBD, it seemed like you were implying that you had sort of multiple targets per program picked, auxiliary targets. I was wondering if you also were thinking about having an auxiliary target for renal transplant and what proportion of the organs that might represent as well. So I'm going to ask Jason to speak to this because in Jason, we recruited someone that did some of the fundamental work on Tregs, but for kidney transplant, about 20% have mismatches, and that means that there is a substantial population of patients who are getting an HLA-A2 mismatch that our That's the beauty of this system.

And your net today.

Between MFS and IBD.

And like.

You were implying that you had sort of multiple targets per program kick on.

And really really target I was wondering.

If you also we're thinking that.

And on selling or and target.

Poor renal transplant, and what proportion of the Oregon and that might represent as well.

So I'm going to ask Jason to speak to this because and Jason we recruited some of them that did some on the fundamental work on on T. Regs.

And the for the kidney transplant is about 20% mismatches.

And that means that there is a substantial population of patients who are getting a neat Chile <unk> mismatch.

And car T Reg can be.

Taken two bites GPS to find day actually to and activate it no matter, what the author mismatches on and the renal transplant message beauty of this system, but Jason do you want to talk about our general strategy for T. Rex.

Alexander D. Macrae: But Jason, do you want to talk about our general strategy for Treg? Sure. Thanks, Andy.

Sure. Thanks Sami.

Jason D. Fontenot: Yeah, I mean, I think one of the things that's critical to keep in mind here is that Sangamo is really, really pioneering this effort. The work that we're talking about is the first opportunity to test engineered CAR Tregs in humans, and the opportunity to use the A2 mismatch presented a very kind of elegant way to approach tissue targeting because we know that the A2 molecule will only be present on the transplanted kidney by virtue of the design of the trial. So that's the real reason for using the A2 approach.

I mean, I think one of the things that.

And that's critical to keep in mind here and size.

Sangamo has really really pioneering this effort.

And the work that we're talking about.

The first opportunity to test engineered car T regs and human.

And so the opportunity to use the <unk> mismatch and <unk>.

And just a very kind of elegant way to approach.

On the tissue targeting because we know that the the <unk> molecule will only be present on the transplanted kidney by virtue of the design of the trial. So that's that's the real reason for using the <unk> approach, but I think one of the really exciting things about the car T Reg platform, especially.

Jason D. Fontenot: But I think one of the really exciting things about the CAR T-REG platform, especially in regards to the subject of your question, which is the antigens that we pick for CAR targeting, is that we are able to pick targets that, as I highlighted, are connected to the disease etiology or simply just tissue-specific targets. And that's a real advantage for us versus some of the problems that we see in the core T field and oncology, where identifying targets that are unique to the cancer has become a real challenge.

And regards to the subject of your question, which is the the antigens that we pick a car targeting is is that we are able to pick targets that as I highlighted.

And are connected to the disease etiology or simply just tissue specific targets.

And that's a real advantage for us versus some of the problems that we see and and the car T field on oncology, where identifying targets that are unique to the cancer has become a real challenge.

Jason D. Fontenot: And so we're able to either use something that is connected to the disease pathology or simply find something that is an antigen that's expressed in the tissue where we want the Tregs to localize. And as we're gaining a huge amount of knowledge and understanding by advancing the TX200 program, we intend to build upon that knowledge in refining which targets we pick for future programs. And so that's why we keep the opportunity to pivot to different targets by advancing multiple targets in each of these.

And so we were able to either use something that is connected to the disease pathology or simply find something that is a and antigen thats expressed in the tissue, where we want the T regs to localize too and as you know the.

And we're gaining a huge amount of knowledge and understanding by advancing the TX 200 program and we're going on and we intend to build upon that knowledge and refining which targets we pick for future programs and so that's why we're keeping the opportunity to pivot to different targets by advancing multiple park.

And each of these indications.

Operator: Got it, and just a quick follow-up on that and the CEDFAS study and follow-up on a previous question. As we think about the two secondary endpoints that you have on slide 12, especially the ability to reduce immunosuppressive therapy, given that the regimens, I guess, can vary from center to center, how will we quantify reductions in immunosuppressive therapy as part of this protocol? The reason I'm asking is that it's my assumption that this might be the first needle to move. Is that a correct assumption?

Got it and.

Just a quick follow up on that and the steadfast study and following up on a previous question as we think about the two secondary endpoints that you have.

On slide 12.

And especially the ability to produce immunosuppressive therapy.

And given that the parent debt the regimen and I guess can vary from center to center.

How will we quantify reductions and immunosuppressive therapy as part of this protocol, especially and the reason I'm asking is it's my assumption that this might be the first needles and Luke is that a correct assumption.

Or could acute graft rejection and generate a first of the efficacy signals between debt Chipotle.

Alexander D. Macrae: Or could acute graft rejection generate the first of the efficacy signals between those two bullets? The advantage of modern medicine is that patients rarely lose their kidneys. The way they avoid that is with immunosuppression, which comes with all kinds of health consequences. This is a cutting-edge, state-of-the-art study that has to allow a normal standard of care by the investigators. Of course, we would love everyone to do the same thing, but the study would not progress without that. And so we will gather information from this. We'll gather information from renal biopsies.

The advantage of modern medicine is patient rarely lose their kidney.

The way the way they are poised on is with immunosuppression, which comes with all kinds of health consequences.

Suez cutting edge state of the art study.

Has to alone.

On normal standard of care by the investigators.

Of course, we would love that and we wanted to do the same thing but.

Studying good not progressed with that.

And so we will conquer the information from this school counter information from renal biopsies.

Alexander D. Macrae: We'll understand more about the Tregs. We'll watch what they do, what happens when they reduce their immunosuppression, and we'll use that to design the next study and the next part of this program.

We'll understand the T ranks, we'll watch what they do if and when and what happens when the reduce the immunosuppression and we'll use that to design. The next study and the next part of this program.

Operator: Thanks for taking the questions. Thank you. Our next question comes from the line of Ben Burnett from Stiefel. Your line is now open. Hi, this is Kaylee Brizon on behalf of Ben Burnett.

Got it thanks for taking the questions.

Thank you our next.

And it comes from the line of Barnett from Stifel. Your line is now open.

Hi, This is Kelly breeze on for Ben Burnett and thanks for taking our question and just had one quick one about T. X 200, I was just wondering if you could provide any additional color about the starting doses that we may expect to see at the first look at data on.

Operator: Thanks for taking our questions. We just had one quick one about TX200. I was just wondering if you could provide any additional color about the starting doses that we may expect to see at the first look at data. We haven't talked about the doses yet. We're going to do three doses, and it will be some number of cells that we will talk about later, but we haven't said anything up to now.

And that if you could read anyway right.

We haven't talked about the doses, which will be some.

And we're going to three doses and there will be some number of sales that we will talk about later, but we haven't said anything up to know.

Operator: Okay, awesome. Thanks for taking our questions. Thank you. Our next question comes from the line of Andreas Argyrides from Wedbush Securities. Your line is now open.

Okay Awesome. Thank you for taking my question.

Thank you. Our next question comes from the line of interest are derived from Wedbush Securities. Your line is now open.

Operator: Good afternoon, and thank you for taking our question. This is Andreas Hans for Liana Masatos. With regard to the ASGCT abstracts, have the respective partners selected a lead candidate yet from the library generated, and when might we see these assets enter the clinic?

Good afternoon, and thank you for taking on our question. This is on drives on for Ravi on them and <unk>.

And with regards to the GCT abstracts have their respective partners selected a lead candidate yet from the library generated and when might be see these assets enter the clinic. Thanks.

I will flow to our partner program such stem to our partners to talk about we we work closely with them, we sit and steering committees with them and so but but the goose of the relationship is for them to talk about it.

Operator: For our partner programs, it's down to our partners to talk about them. We work closely with them, we sit on steering committees with them, but the rules of the relationship are for them to talk about it.

Operator: Okay, so how should we or investors look at the abstracts being presented from a scientific standpoint?

Okay. So how should we how should we are investors.

Look at the day, the abstracts being presented from a scientific standpoint.

Alexander D. Macrae: From a scientific standpoint, I think it's very fair, but I want to assure you that our partners are excited by the progress of the CNS assets, and we look forward to speaking more about them in the future.

From a scientific standpoint, I think is very fair and but I want to assure you that our partners are excited by the progress of the CNS assets and we look forward to speaking more about them and the future.

Operator: Okay, thank you. I appreciate it.

Okay. Thank you I appreciate it.

Operator: Thank you. Our next question comes from the line of Debjit Chattopadhyay from Guggenheim Securities. Your line is now open. Hi, guys. Thanks for taking my question. This is Aaron from DevJet. So, we noticed from the Fabry clinical trials entry that the patients must have cornea vericulite, acroparasthesia,

Thank you. Our next question comes from the line of and sheet.

She was trying to find me from Guggenheim Securities. Your line is now open.

Hi, guys. Thanks for taking my question Erinn on the bad debt. So we know.

Notice from the Fabry clinical trials and treated the patients must have cornea particular AG.

Crow Paresthesia and hi, Joseph Angiokeratoma be included so can you tell us any estimate of the percentage of fabry patients with those and then and why those particular symptom.

Operator: Can you tell us any estimate of the percentage of SABRI patients with those symptoms and why those particular symptoms?

Alexander D. Macrae: So Bettina, this feels like one for you. Yes, thank you, Sandy. So, as you rightly point out, clinicaltrials.gov, we have listed the inclusion and exclusion criteria for our FABRI-STAR study. And the symptoms you listed, such as corneal verticillata, agloparesthesia, anhydrosis, endocaritoma, these are symptoms that are classic in FABRI patients. They are, Patients need to have at least one of those symptoms, if not more, to be able to be enrolled in the study.

So patina of this feels like one for you.

Yeah. Thank you Sandy.

So on.

And you rightly point out clinical trials that we have listed the inclusion and exclusion criteria for all fabry.

Hi, Savi and.

The symptoms you listed so currently on Latam.

Yeah on hydro is underpinning some of these symptoms.

Symptoms that are classic hubs.

B patients.

They are.

Need to have at least one of those symptoms if not more to be able to be enrolled into the study and that is to ensure that beyond the documented diet.

Bettina M. Cockroft: And that is to ensure that beyond the documented diagnosis of FABRI disease, we are enrolling patients who do have the underlying condition. Okay, great. Thank you. Thank you. At this time, I'm not asking any further questions. I would like to turn the call back over to Sandy Macrae, CEO, for closing remarks. Thanks so much. This is Erin. I'll take that.

Fabry disease, we are enrolling patients and have the underlying condition.

Okay, great. Thank you.

Thank you at this time line is showing no further questions I would like to turn the call back over to Sandy Macrae.

Oh for closing remarks.

Thanks, So much this is Aaron I'll take that so thanks on sedan everyone.

Erin Feingold: So thanks once again, everyone, for joining us today. And for your questions, we look forward to keeping you updated on our future developments. Have a great afternoon. This concludes today's conference call. Thank you for participating. You may now disconnect.

Everyone for joining us today and for your question and you look forward to keeping you updated on our teacher development hungry and Australian.

This concludes today's conference call and thanks for participating you may now disconnect.

Operator: Good night.

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