Sangamo Therapeutics, Inc.

Thank you all for standing by and welcome to the Sangamo Third Quarter 2021 teleconference call. All participants are in a listen-only mode until the question and answer session of today's conference. To ask a question over the phone by that time, you may press the star key followed by the number one. Please also note that today's call is being recorded. I'll now turn the call over to your host, Aaron Feingold, Head of Corporate Communications. Ma'am, you may now begin.

Good morning, and thank you for joining us today. With me this morning on this call are several members of the Sangamo Executive Leadership Team, including Sandy McRae, Chief Executive Officer, Mark McClung, Chief Operating Officer, Pratusha Durrababu, Chief Financial 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 under 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 therapeutic and commercial potential of our product candidates. The anticipated plans and timelines of Sangamo and our collaborators for conducting clinical trials and presenting clinical data, execution of our corporate strategy, advancement of our product candidates, our revised 2021 financial guidance, and other statements that are not historical facts. Actual results may differ materially from what we discussed today. 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, as supplemented by our quarterly report on Form 10-Q for the fiscal quarter ended September 30, 2021. The forward-looking statements stated 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. 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 McRae.

Thank you, Aaron, and good morning to everyone on the call. This is such an important moment for Sangamo, as we share clinical data and business updates across several programs, demonstrating that we have three important assets in or progressing towards late-stage development. Our gene therapy portfolio is advancing with accumulating safety and efficacy data in our Fabry and Haemophilia A programs. Additionally, we are delighted with the preliminary proof of concept data demonstrating the clinical potential of our genome engineering zinc finger technology in sickle cell disease. This morning, we announced preliminary clinical data from our phase one two-star study, evaluating Isoralgagene, Sivoparvivec, or ST920, our Fabry disease gene therapy product candidate. Data from this important study were evaluated from the four patients in the first two cohorts, those levels 0.5E13 and 1E13 BG per TIG. As of the cutoff date, September 17th of this year, These encouraging results showed that for the first four patients, ST920 was generally well tolerated. There were no treatment-related adverse events higher than grade one and no treatment-related serious adverse events. No patients experienced liver enzyme elevations or required steroid treatment. All four patients exhibited above normal alpha-gallate activity. which were maintained for up to one year for the first patient treated and through 14 weeks for the most recently treated patients. Levels ranged from 2 to 15-fold above normal levels at last measurement as of the cut-off date. Interestingly, the first three patients still reported improvements in ability to sweat, a primary and common Fabry disease symptom that limits exercise tolerance for the patient. ERT withdrawal is now complete for one patient and is planned for the other patient on ERT, based on the stability of their alpha-galli activity following treatment. Based on these data, we have initiated phase 3 planning. The fifth patient in the study, who is the first patient in the third dose cohort at 3E13, was recently dosed. The sixth patient is currently in screening, also for the third dose cohort. and we expect to provide updated results throughout 2022 and present these data at a medical meeting. This morning, we also announced that preliminary proof-of-concept results from the Phase 1-2 precision study of SAR 445136 and investigational zinc finger nucleus gene-edited cell therapy in patients with sickle cell disease will be presented at ASH. This program is partnered with our friends at Sanofi. The data in the abstract shows that as of June 25th, 2021 cutoff date, none of the four patients treated required blood transfusions, post-engraftment through 65 weeks of follow-up for the longest treated patients. The four treated patients all experienced increases in total hemoglobin, fetal hemoglobin, and percent F cells. No adverse events or serious adverse events related to treatment were reported as of the cutoff date. Further data will be provided in a poster presentation at ASH on December the 12th. Sangamo and Sanofi are continuing to advance the sickle cell disease program. The company's recently obtained manufacturing requirements guidance from the FDA in preparation for potential further clinical studies. Separately, we in Sanofi made the business decision to cease development for the beta thalassemia indication and allowing us to focus resources on the sickle cell disease program. Moving now on to our haemophilia aid program partnered with Pfizer, we announced this morning the updated follow-up results from the Phase 1-2 ALTA study of durotocogene phytocarvavec will be presented at ASH. For the four patients in the highest dose cohort who have been followed for at least 104 weeks as of May 19, 2021 cutoff, mean factor rate activity was 30.9% at week 104 as measured by chromogenic assay. In this cohort, annualized bleeding rate was zero for the first year after treatment and 0.9 throughout total duration of follow-up. As demonstrated in this study, the gene therapy was generally well tolerated in patients with severe haemophilia A. Further data will be provided in a poster presentation at ASH on December the 12th, 2021. We and Pfizer also announced that some of the patients treated to date in the phase three of fine trial experienced factor VIII levels greater than 150% following treatment. To date, none of these patients have experienced thrombotic events, and some have been treated with direct oral anticoagulants to reduce thrombotic risk. Out of an abundance of caution, Pfizer voluntary paused screening and dosing of patients in the trial in order to implement a protocol amendment which will provide guidelines for clinical management of elevated factor VIII levels. On November 3, Pfizer was informed that the FDA has put this trial on clinical hold. Pfizer and Sangamo are committed to resuming patient dosing as soon as possible. We continue to believe that this gene therapy will represent an important treatment option for patients with haemophilia A. The next step is to share the proposed protocol amendment with health authorities and respond to the clinical hold, after which the companies will be able to provide updated timing for the trial. Turning now to our kidney transplant programme, we have now enrolled the first patient in our Phase 1-2 Steadfast Study, evaluating TX200, our wholly-owned autologous CAR Treg cell therapy candidate. We believe that this is the first in-human CAR Treg study and that this field is growing with much excitement as a promising approach for challenging autoimmune conditions. In this study, similar to other genetically engineered cell therapy approaches, patients will undergo a lookup procedure from which their Treg cells will be isolated, engineered, and then cryopreserved. The HLA-A2 negative patient will subsequently undergo kidney transplant and, following a recovery period, will receive their personalized TX200 therapy. We expect to dose the first two patients in this study by the middle of 2022, following their kidney transplants. We continue to open sites and screen patients. We believe this proof of concept study may represent an important treatment for patients undergoing renal transplant and will help us understand CAR Treg biology in humans, as well as advanced process development knowledge. We hope that this study establishes the foundation for a portfolio of wholly owned CAR Treg therapies for autoimmune indications. Finally, I'm delighted to share that Mark McClung has been appointed as Sangamo's Chief Operating Officer effective November 1st. Mark's expanded role as COO is an important organisational step for Sangamo, which will support the multiple advancing wholly owned and partnered programmes. We look forward to Mark's continued leadership as we continue to build the capabilities to bring genomic medicines to patients and to the marketplace. Everyone at Sangamo is thrilled about this clinical momentum, and we look forward to presenting updated Fabry results throughout 2022, as well as working with our collaboration partners and investigators to present the ASH data in December. And with that, I'll turn the call over to Patricia for a financial update. Patricia.