Taysha Gene Therapies, Inc.

Welcome to the Tayshia Gene Therapy's Full Year 2020 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode. Following the management's prepared remarks, we will hold a brief question-and-answer session. As a reminder, this call is being recorded today, March 3, 2021. I will now turn the call over to Dr. Kimberly Lee, Senior Vice President of Corporate Communications and Investor Relations. Please go ahead.

Thank you, and good morning, and welcome to Tayshia's full year 2020 financial results and corporate updates conference call. Joining me on today's call are R.A. Session II, Tayshia's president, CEO, and founder, Dr. Suyash 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 full year ended December 31, 2020. 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 fact 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, March 3rd, 2021. TASHA undertakes no obligation 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. I would 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 first corporate update and financial results conference call. We hope you and your family continue to remain safe and healthy. In the past year, we have made significant progress on our corporate initiatives. I will elaborate on some of our key achievements and upcoming expected milestones. And then we will turn the call over to Suyash and Cameron for updates on our pipeline development and financial results, respectively. 2020 was a highly successful and foundational year for the company, marked by many significant milestones. Since March 2020, we have raised gross proceeds of $307 million, which included approximately $181 million in gross proceeds from the completion of a successful IPO last September that included participation from a significant number of high-quality healthcare-focused institutional investors, which increased our visibility within the broader investment community. This was one of the fastest seed to IPOs in biotech history, which we consider a reflection of the team's commitment and our investors' confidence in our innovative approach to gene therapy. Central to TASHA's success is our strategic collaboration with the UT Southwestern Medical Center Gene Therapy Program, one of the premier academic medical centers in the world. We hold an exclusive worldwide royalty-free license from UT Southwestern to discover, develop, and commercialize gene therapies for our pipeline. Our collaboration with UT Southwestern is led by Dr. Steven Gray and Burge Manassian. Dr. Gray is the Associate Professor in the Department of Pediatrics at UT Southwestern and an expert in the development of AAV-based gene therapies for CNS disorders. Dr. Manassian is the Division Chief of Child Neurology and faculty at the Children's Medical Center Research Institute at UT Southwestern and a seasoned clinician in the diagnosis, management, and treatment of rare pediatric neurological diseases. Through our partnership, we are advancing a deep and sustainable pipeline that currently consists of 25 gene therapy product candidates. Our portfolio targets monogenic diseases of the central nervous system across three distinct franchises, neurodegenerative diseases, neurodevelopmental disorders, and genetic epilepsy. By leveraging synergies across our programs, we are well positioned to advance our current pipeline while actively promoting developing our novel next-generation platforms to expand the limits of gene therapy into indications that are currently unaddressable with available technologies. We are complementing our efforts to expedite the development of our current programs by working closely with regulatory authorities, and we have already secured rare pediatric disease and orphan drug designations from the FDA for six product candidates, TASHA 101 for GM2 gangliosidosis, TASHA-102 for Rett syndrome, TASHA-103 for SLC6A1, haploid insufficiency, TASHA-104 for SIRT1-associated Lee syndrome, TASHA-105 for SLC13A5 deficiency, and TASHA-118 for CLN1 infantile badness disease. Our relationship with patient advocacy organizations and research foundations remain very important to us. As such, we continue to establish further strategic partnerships, including with Invitae and AllStripes, to support access to genetic testing and earlier diagnosis of patients with CNS disease, and to inform the understanding of the natural history, disease burden, and patient diagnostic journey. We believe this will also accelerate our efforts to enroll patients for our clinical trials. Part of our approach to accelerate the development of our pipeline is integrating our R&D and GMP manufacturing capabilities to sufficiently meet the clinical demand of our extensive portfolio. Our three-pillar approach to manufacturing includes our partnership with UT Southwestern, where we have access to a 500-liter GMP-compliant manufacturing suite for early-phase clinical and IND-enabling toxicology materials, and our manufacturing partnership with Catalan for early-phase and pivotal clinical supply. The third pillar is the establishment of our internal 187,000 square foot commercial scale GMP compliant manufacturing facility with multiple production suites and an initial capacity of 2,000 liters to support preclinical through commercial GMP manufacturing for our pipeline. We believe the addition of an internal facility will enable us to drive efficiencies and scalability across our manufacturing supply chain. in order to meet the potential demand of our multiple concurrent programs. We expect to initiate construction on this facility later this year. As part of our manufacturing strategy, we are also spending time and effort on CMC characterization, potency assays, titering assays, and other associated lab work. Our employees are foundational to our ability to quickly advance the development of gene therapies. Keisha is growing at such an incredible pace. that we have more than doubled our employee base to approximately 80 in just the last three months. Moreover, we expect to expand the team to approximately 150 employees by year-end to support our robust development and corporate initiatives. We are privileged to have our efforts augmented through partners and advisors who are trailblazers in the gene therapy space. Beyond our strategic collaboration with Dr. Gray and Monastian, We are fortunate to be advised by an independent, internationally renowned scientific advisory board with academic credibility and significant industry experience, and a seasoned board of directors consisting primarily of industry-leading gene therapy executives and scientists. With their support, we believe we are uniquely positioned for sustained success as we further our R&D initiatives and advance our next-generation technology platform. We anticipate a transformational year as we expect to report first-in-human clinical data for TASHA 101 in GM2 ganglion cytosis and launch four candidates into Phase I-II studies following acceptance of their INDs or CTAs. We have currently advanced an additional five therapies into IND or CTA-enabling studies and have initiated four new programs into preclinical development. Importantly, we expect to advance our next generation technologies to optimize key components of AAV-based gene therapy. We look forward to leverage our capabilities to pioneer novel approaches to address vector capacity and to continue to innovate as it pertains to payload design. Lastly, we will continue to evaluate opportunities to maximize the value of our existing pipeline. I will now turn the call over to Suyush to provide an update on our R&D initiatives. Suresh, please go ahead.