Sangamo Therapeutics, Inc.

Good day and thank you for standing by. Welcome to the Sangamo Therapeutics fourth quarter and full year 2021 conference call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you'll need to press star then one on your telephone keypad. Please be advised, today's conference may be recorded. If you require operator assistance during the call, please press star then zero. I'd now like to hand the conference over to your host today, Erin Feingold, Head of Corporate Communications. Please go ahead.

Good afternoon, and thank you for joining us today. With me this afternoon on this call are several members of the Sankmo Executive Leadership Team, including Sandy McRae, Chief Executive Officer, Mark McClung, Chief Operating Officer. Pratusha Dharibabu, 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 Sections 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 the therapeutic and commercial potential of our product candidate, the anticipated plans and timelines of Sangamo and our collaborators for initiating and conducting clinical trials and presenting clinical data, execution of our corporate strategy, advancement of our product candidates, our initial 2022 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 31st 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.

Thanks, Erin. And good afternoon to everybody on the call. I'd like to start by saying that 2021 was a significant year for Sangamo as we continue to advance the development of genomic medicines for patients across multiple therapeutic areas using our innovative technologies. We're very pleased with our progress despite the challenges of the second year of the pandemic. We're advancing potentially transformative genomic medicines in the clinic and strategically using our R&D capabilities to pursue indications of unmet need. These efforts are supported by our manufacturing infrastructure, including in-house AAV and cell therapy facilities. Our collaboration partners also help us drive toward our mission to deliver on the promise of genomic medicine. And we believe that this progress positions us as well to generate long-term value for our shareholders. In 2021, we executed upon our strategy with several important achievements. First, we and our partners advanced our three-league programs while presenting compelling clinical data. Starting with our wholly owned Phase 1-2 Fabry disease programme, we presented updated data at the World Symposium earlier this month. We're encouraged by the safety and efficacy data we have seen to date. And most importantly, the patients in the study have reported they are feeling better. Investigators are observing improvement in some of the most challenging symptoms, including ability to sweat in the first three treated patients. With the recent changes in the Fabry competitive landscape, we believe we are in a leading position. In the second half of this year, we plan to present additional updated Phase 1-2 data. We're actively planning for a Phase 3 study, including discussions with health authorities, patient advocacy groups, and investigators. We're also delighted by the emerging Phase 1-2 sickle cell disease data presented at ASH in December, showing no treatment-related adverse events in the four treated patients, improvement across several biomarkers, and most importantly, clinically significant reduction in painful sickling crisis. We anticipate that the next four patients treated in the study will be dosed with a product candidate manufactured using improved methods that have been shown in the internal experiments to increase long-term progenitor cells. We expect to complete dosing of these patients in the third quarter of this year. Transition planning of the program from Sanofi to Sangamo is going well, and we are energized to have this asset back in our hands soon as we assess the best way to move the program forward for patients, be that on our own or with a potential partner. Finally, we're encouraged by the follow-up data presented at ASH last year from our Haemophilia A program partnered with Pfizer. Updated Phase 1-2 results show sustained bleeding control in the highest dose cohort through two years following gene therapy. Regarding the phase three study, Pfizer has announced that it hopes to obtain agreements with the health authorities to resume their final trial in the first half of 2022. The trial was previously paused when some of the patients experienced factor VIII activity greater than 150% following treatment. Pfizer is currently in the process of submitting a protocol amendment to health authorities in the countries where this trial has been conducted and preparing responses to the FDA clinical hold. Over 50% of the patients have been enrolled in the phase three affine trial. Second, we're progressing our preclinical candidates based on our second generation technologies, CAR Tregs for autoimmune disease and zinc finger transcription factors for neurological disorders. We have enrolled and expect to dose soon the first patient in our lead CAR Treg program, where we are evaluating TX200 for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplant from a living donor. We believe that this will be the first patient ever to be dosed with a CAR Treg therapy and that we are in a leading position with several companies following us into this very promising area. We believe that our expertise across multiple technology platforms, robust cell therapy infrastructure supported by our manufacturing facilities and genomic engineering capabilities and internal strategic and operational synergies comprise a differentiated CAR T-REG platform from which we can potentially offer patients advanced genomic medicines. In addition to our proof of concept study of TX200, we are progressing our preclinical allogeneic renal transplant rejection study, as well as inflammatory bowel disorder and multiple sclerosis programs, including presenting in the first preclinical data from our allogeneic IL-23R CAR-T reg candidate in IBD last year. And finally, with regard to our zinc finger protein transcription factor technology in treating CNS disorders, In addition to our partner programs with Biogen, Novartis, Takeda, and Pfizer, we're advancing multiple internal programs. Third, we continue to hone our differentiated genome engineering platform, including improving the specificity, precision, and efficiency of our cores and finger proteins. We're also progressing our capabilities from nucleases to repressors, activators, and even base editors, and are excited about our progress. We see Cynomos capabilities as representing a one-stop shop for a range of genomic engineering capabilities that are designed to be applied therapeutically. Fourth, we continue to work diligently with our collaborators, supporting the advancement of our partner programs in the clinic, while driving research efforts for preclinical programs for which we receive reimbursement from our partners. These partnerships have been a key component of our development strategy and continue to drive Maui for Saigon. We believe that the buy-in from Pharma validates our mechanistic approach across a range of advanced modalities and as it enables us to benefit substantially from our partners domain expertise to develop high quality therapeutics for patients. The capital provided by our partnerships helps to advance our internal pipeline of assets. while providing our partner programmes with the resources needed to advance the development of these potentially transformative therapies more quickly. Fifth, we completed and brought online our cell therapy manufacturing facilities in Brisbane and Valbonne, and now have operational AAV and cell therapy facilities in-house. We believe these facilities provide many strategic advantages, including flexibility in control, capacity to support our R&D needs, process expertise, geographic diversification, and that supports supply chain resilience and a deep intellectual property portfolio. Six, we believe that we have a strong financial position to take us through our key upcoming catalysts. Our diverse and accomplished leadership team and our talented employees are passionate about our mission and have enabled our multiple 2021 accomplishments, setting us up for what we expect to be a strong 2022. I am very grateful to my leadership team and all my Samuel colleagues for their dedication and hard work in a second challenging year of the pandemic. And with that, I'd like to turn the call over to our head of development, Rob Schott, who will discuss the data from our clinical programs in more detail.