Passage Bio, Inc.

Thank you for holding. Good morning and welcome to the PassageBio second quarter 2021 financial and operating results conference call. At this time, all participants are in a listen-only mode. Following the formal remarks, we will open the call up for your questions. Please be advised this call is being recorded at the company's request. At this time, I would like to turn it over to Stuart Henderson, Vice President of Investor Relations and Strategic Finance. Stuart, please begin.

lines to topics we plan to discuss today. This release is available on the PassageBio website at investors.passagebio.com under the Press Releases and Statements section. On today's call, Bruce Goldsmith, President and Chief Executive Officer, will review our second quarter 2021 financial results and discuss recent business highlights. Eliseo Salinas, our Chief R&D Officer, will review our key clinical updates, and Jill Quigley, Our chief operating officer will also be available for the Q&A portion of the call. Before we begin, please note that today's call may include a number of forward-looking statements, including but not limited to comments on our expectations about timing and execution of anticipated milestones, including the initiation of clinical trials and the availability of clinical data from such trials, our expectations about our collaborators' and partners' ability to execute key initiatives, the ability of our lead product candidates to treat their respective target CNS disorder, manufacturing plans and strategies, trends with respect to financial performance and cash flows, the company's ability to fund research and development programs, impacts of the COVID-19 pandemic on the company's operations, and its ability to manage costs along with uses of cash and other matters. These forward-looking statements are based on assumptions that are subject to risks and uncertainties that could cause the company's actual results to differ significantly from those suggested by these statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. Please refer to the company's filings with the SEC for information concerning risk factors that could cause its actual results to differ materially from expectations, including any forward-looking statements made on this call. Except as required by law, the company disclaims any obligation to publicly update or revise any forward-looking statements to account for or reflect events or circumstances that occur after this call. It is now my pleasure to pass the call over to CEO Bruce Goldsmith. Bruce?

Thanks, Stuart, and thank you all for joining us this morning. I will begin by centering my remarks around our four strategic pillars that we believe differentiate our company. These pillars are our strong partnership with Penn's Gene Therapy Program, or GTP, which is led by our Chief Scientific Advisor, Dr. Jim Wilson, our broad and robust pipeline, our integrated manufacturing supply chain, and our solid corporate foundation, which includes a highly experienced team and a strong cash position. These pillars provide the framework for where and how we continue to focus our efforts as we develop transformative medicines for patients with CNS disorders and establish our company as a leader in genetic medicines. For our partnership and pipeline, I am excited to report that we have amended our existing strategic collaboration and license agreement with GTP to broaden the scope to include large CNS diseases. We view this as a significant inflection point in the company's evolution. We also believe that this continues to strengthen our positive and productive collaboration with Jim Wilson, his dedicated expert team, and the University of Pennsylvania. Under the expansion into larger populations, the new exploratory research programs, which will be guided collaboratively by GTP and PassageBio, will support the identification and evaluation of biological targets and generate preclinical proof of concept data for these targets. we will be able to evaluate several targets and technologies in parallel to enable quick decision-making. Once a target has reached proof of concept, we may then exercise an option to advance to development of a new genetic medicine for the underlying condition, but are under no obligation to exercise that option. We are initiating this effort focusing on Alzheimer's disease and temporal lobe epilepsy, with the opportunity to expand to additional large CNS indications upon mutual agreement. These research programs and our ability to select candidates across a wide range of CNS indications beyond rare monogenic conditions will bolster our already robust pipeline and provide greater optionality for program advancement. In our agreement with GTP, we have the option to license 17 total programs. So far, we have exercised seven of these options in rare monogenic diseases, and we have 10 remaining. In addition to the expansion into large indications, we maintain our current focus on rare monogenic disorders and may exercise additional options in this area as well. When exercising our remaining 10 options, we will continue to do so in a data-driven manner, factoring in patient need, product differentiation, and the ability to de-risk clinical development programs. For our manufacturing capabilities, I'm happy to report that we have met our goal to open our new gene therapy research and development lab called Princeton West in Hopewell, New Jersey. With this facility, we have enhanced our analytical development, clinical product testing, and assay validation capabilities. State-of-the-art analytics are essential for optimizing vector production and appropriately characterizing our gene therapy products as we advance clinical development, and eventually move to commercial-scale production. Our strength across these first three pillars, partnership, pipeline, and manufacturing, is underpinned by our solid corporate foundation, including the world-class leaders we have successfully recruited. We are proud of the caliber of talent we have attracted to Passage Bio, as illustrated in our recent announcement of new executive appointments. Maria Tornson, joins us as Chief Commercial Officer and is responsible for leading and building out the company's commercial organization and strategy, including determining product positioning and paths to commercialization. Simona King joins us later this month as Chief Financial Officer and will be responsible for finance, accounting, tax, treasury, investor relations, functions, and information technology. Mark Forman joins us as chief medical officer and is responsible for leading the company's translational and clinical development efforts. We also have a strong board of directors supporting and guiding our company. The recent addition of Dr. Darrell Porter, who brings tremendous experience in corporate strategy, product development, and commercialization, is an example of the kind of talent and expertise we're able to call upon at the board level. As we build our company around our four strategic pillars, we are hyper-focused on the strong execution of our three lead clinical development programs in rare CNS disorders for which there are limited or no therapeutic options. We remain on track despite ongoing delays due to COVID-19 to deliver on several important clinical milestones. These include dosing the first patients in our Uplift-D study for the treatment of frontal temporal dementia with granulone mutations, and Galaxy for the treatment of Krabbe disease in the third quarter of 2021. Reporting initial safety and 30-day biomarker data for the first cohort of patients in our IMAGINE 1 trial for the treatment of infantile GM1 in the fourth quarter of 2021. And reporting initial safety and 30-day biomarker data for the first cohort of patients in our UPLIFT-D and Galaxy trials in the first half of 2022. Our momentum and execution are supported by a strong balance sheet with $408 million in cash, cash equivalents, and marketable securities at quarter end. I will now turn it over to our Chief R&D Officer, Eliseo Salinas, who will discuss our clinical programs in more detail.