Taysha Gene Therapies, Inc.

Welcome to the Tayshia Gene Therapy's second quarter 2021 financial results and corporate update conference call. At this time, all participants are in a listen-only mode. Following management's prepared remarks, we will hold a brief question and answer session. As a reminder, this call is being recorded today, August 16, 2021. I will now turn the call over to Dr. Kimberly Lee, Senior Vice President of Corporate Communications and Investor Relations. Please go ahead.

Good morning and welcome to Tayshia's second quarter 2021 financial results and corporate update conference call. Joining me on today's call are Ari Session II, Tayshia's president, CEO, and founder, Dr. Suresh Prasad, chief medical officer and head of R&D, and Cameron Alam, chief financial officer. After our formal remarks, we will conduct a question and answer session and instructions will follow at that time. Earlier today, Tayshia issued a press release announcing financial results for the second quarter ended June 30th, 2021. A copy of this press release is available on the company's website and through our SEC filings. Please note that on today's call, we will be making forward-looking statements, including statements relating to the safety and efficacy and the therapeutic and commercial potential of our investigational drug candidates. These statements may include the expected timing and results of clinical trials for our drug candidates, and the regulatory status and market opportunity for those programs, as well as Tayshia's manufacturing plans. This call may also contain forward-looking statements relating to Tayshia's growth and future operating results, discovery and development of drug candidates, strategic alliances and intellectual property, as well as matters that are not historical facts or information. Various risks may cause Tayshia's actual results to differ materially from those stated or implied in such forward-looking statements. These risks include uncertainties related to the timing and results of clinical trials and preclinical studies of our drug candidates, our dependence upon strategic alliances and other third-party relationships, our ability to obtain patent protection for our discoveries, limitations imposed by patents owned or controlled by third parties, and the requirements of substantial funding to conduct our research and development activities. For a list and description of the risks and uncertainties that we face, please see the reports we have filed with the Securities and Exchange Commission. This conference call contains time-sensitive information that is accurate only as of the date of this live broadcast, August 16, 2021. Taysha undertakes no obligations to revise or update any forward-looking statements to reflect events or circumstances after the date of this conference call, except as may be required by applicable securities law. With that, I'd now like to turn the call over to our President, CEO, and Founder, R.A. Session II.

Thank you, Kim. Good morning and welcome, everyone, to our second quarter financial result and corporate update conference call. Taysha continues to make significant progress on several key clinical manufacturing and strategic corporate initiatives, which were highlighted at our recent R&D and Manufacturing Investor Days, as well as our recent press release announcing our non-dilutive financing with Silicon Valley Bank. I will elaborate on some of our recent key achievements and review the expected milestones for the remainder of 2021. Following this, I will turn the call over to Suyesh and Cameron for updates on our pipeline developments and financial results respectively. At our R&D day held in June, we presented positive data on a number of our key development programs. These data included phase 1 slash 2 visual acuity data for TASHA 120 in patients with giant axonal neuropathy, or GAN, genotypic-specific MEKP2 expression data for TASHA 102 in Rett syndrome, natural history data in GM2 gangliosidosis, preclinical data for TASHA 118 in CLN1, natural history data in CLN1 disease, along with clinical patient phenotype data in SIRF1-associated leaf syndrome. We also disclosed the positive effects of TASHA-105 on seizures and associated deaths in SLC1385 knockout mice, the effects of TASHA-103 on abnormal EEG activity in SLC6A1 knockout and heterozygous mouse models, and preclinical data for TASHA-112 in APBD and TASHA-111 leforin and TASHA-111 malin in leforin disease. Lastly, we disclosed the effects of TASHA-113 on tau expression in MAPT-associated tau apathy, and our two novel approaches for the treatment of Angelman syndrome. Sunyush will review these pipeline developments shortly. Part of our success relies on robust, sustainable, and high-quality manufacturing to support our portfolio, and we are pleased to announce that we have achieved several successful GMP runs that further support our five planned Phase 1-2 clinical trials and numerous INB-CTA-enabling studies. In July, we held a manufacturing day to highlight how our flexible and scalable approach which seamlessly integrates R&D and manufacturing, de-risks our overall portfolio and supports consistent delivery of high-quality clinical material across our broad pipeline. Our three-pillar approach to manufacturing includes dedicated capacity at UT Southwestern, a collaboration with Paragon, a subsidiary of Catalan, and the development of our internal manufacturing facilities. We continue to make progress on the construction of our multi-product facility in Durham, North Carolina, which will have 2,000 liters of capacity. Additionally, the capacity at UT Southwestern is expected to increase from 500 liter to 700 liter scale by the end of the year, which will continue to support our IND enabling and early clinical trial efforts and ensure high-quality, efficient, and accessible production for our pipelines. In order to further our mission in the development of novel gene therapies for the treatment of monogenic diseases of the CNS, we recently became a founding member of the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life science companies committed to discovering, developing, and delivering rare disease treatment. We look forward to working together to expedite the delivery of our transformative therapies to the millions of patients with rare diseases. In the second half of this year, we expect numerous value-creating preclinical, clinical, and regulatory milestones. Recently, we have had a number of meetings with multiple regulatory agencies regarding our Rett syndrome, GM2 gangliosidosis, and CLN1 disease programs as we prepare for IND slash CTA submission. I am pleased to share that we have received positive feedback from these agencies that paved the way for multiple anticipated IND-slash-CTA filings in the second half of this year. For tissue 120, our pivotal stage product candidate in GAN, we anticipate data from the high-dose cohort in the second half of this year and expect to provide a regulatory update by year end. For GM2 gangliosidosis, we remain on track to report first in human safety and HEXA biomarker data in the second half of this year. For our CLN1 program, which currently has an open IMV, we anticipate dosing of the first patient in our Phase 1-2 trial in the second half of this year. Collectively, by year-end, we anticipate that we will have five programs in clinical development. The rapid advancement of our numerous candidates would not be possible without the support of our dedicated and talented team. We recently appointed Mary Newman as Chief Development Officer. She was a former Senior Vice President of Regulatory Affairs at Astellas Gene Therapies, formerly Audentes, and brings over 30 years of experience in translational development, program management, and regulatory affairs. At Tayshia, we now have 155 employees, in addition to 70 colleagues at UT Southwestern across multiple functional areas, including discovery, translational development, GMP manufacturing, and clinical care. The collective expertise and dedication across these teams, including our seasoned board of directors, an independent, internationally renowned scientific advisory board, uniquely positions the expedited development of our gene therapy candidates and our technology platform. We have ambitious corporate objectives planned over the next 12 to 18 months. And we are very pleased to have recently entered into a non-dilutive term loan agreement with Silicon Valley Bank that provides Taysha with up to $100 million in non-dilutive financing at an attractive interest rate that lowers our overall cost of capital, bolsters our cash position, and provides additional financial and operational flexibility. We believe full drawdown of this funding will extend our cash runway to support key value-creating milestones including the release of Phase 1-2 data from the highest dose cohort in GAN and Phase 1-2 data in GM2 gangliosidosis, CLN1 disease, and Rett syndrome, and importantly, a potential regulatory approval for TASHA 120 in GAN without the need for additional financing. We look forward to updating you on our continued progress throughout the remainder of the year, including at our upcoming Virtual Investor Miniseries for our CLN1, Rett Syndrome, and Angel Syndrome programs, where we will feature presentations from key opinion leaders and highlight progress made to date. I will now turn the call over to Suyesh to provide a more detailed update on our R&D initiatives. Suyesh, please go ahead.