REGENXBIO Inc.

Good afternoon and welcome to the Region X BIO first quarter 2021 earnings conference call. At this time, all participants are in a listen only mode. Later, we will conduct the question and answer session and instructions will be given at that time. As a reminder, this conference call is being recorded. I would now like to turn the call over to Mr. Patrick Christmas, Chief Legal Officer of Region X BIO You may begin.

Good afternoon, and thank you for joining us. With us today are Ken Mills, Regenexx Bio's President and Chief Executive Officer, and Dr. Steve Pakola, our Chief Medical Officer. Earlier this afternoon, Regenexx Bio released financial and operating results for the first quarter ended March 31, 2021. The press release reporting our financial results is available on our website at www.regenexxbio.com. Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend, and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance, and involve certain risks and uncertainties. These risks are described in the risk factors and the management discussion and analysis sections of Regenexx BIO's annual report on Form 10-K for the full year ended December 31, 2020, and comparable risk factors sections of Regenexx BIO's quarterly reports on Form 10-Q on file with the Securities and Exchange Commission and available on the SEC's website. Any information we provide on this conference call is provided only as of the date of this call, May 5th, 2021, and we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company or Actual results may differ materially. I would now like to turn the call over to Ken Mills.

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I hope everyone's continuing to stay safe and healthy. On today's call, Steve and I will review our recent progress, advancing and expanding our internal pipeline based on our proprietary NAV technology, discuss future milestones, and VIT is unable to join us today, so I will provide an update of our financial results for the first quarter of 2021. At the end of the call, we'll open the line for questions. To begin, I want to express how encouraged I am by the dedication of our team and what they've shown and the progress we've made for our pipeline of AAV gene therapies for patients. In the first quarter of this year, we've achieved quite a few milestones. We've initiated our first pivotal trial, Atmosphere, to evaluate RGX314, a one-time gene therapy for the treatment of wet AMD. and we look forward to moving towards a potential BLA filing in 2024. We're also evaluating the suprachoroidal delivery of RGX314 using the SCS microinjector, a targeted in-office approach, and we have completed dosing in our second cohort of our ongoing Phase II AV8 trial for the treatment of wet AMDA. Today, we also announced that we have expanded the AV8 trial to include a third cohort, and Steve will walk through further details about that in just a moment. Our team has also made great strides on our rare genetic disease programs. We're pleased with the continued development and advancement of our RGX121 clinical program for the treatment of MPS. We recently provided an update at the World Symposium for our ongoing Phase 1-2 trial of RGX121 in patients up to five years old, and look forward to providing additional data from this trial in 2021, including next week at ASGCT. We also announced today that we've dosed the first patient in a Phase I-II trial in older patients with MPS II, where we hope to gain additional insight into the potential treatment effects of RGX-121. We've recently completed enrollment in the first cohort of our Phase I-II trial of RGX-111 for the treatment of MPS II. And our team has also been working diligently towards an expected IND submission of RGX202 for the treatment of Duchenne muscular dystrophy in mid-2021. Here at Regenexx Bio, we continue to believe in the promise of our NAV technology and remain committed to the developing gene therapies that improve treatment options for people with serious diseases. I'm grateful for the relationships we've forged with physicians, patients, patient advocates, and we're dedicated to supporting them in many other ways as well. We've participated recently in several events sponsored, for instance, by the National MPS Society, including the virtual Race for a Cure this past weekend. And next week, we'll join their annual celebration of MPS Awareness Day. We look forward to connecting with friends and colleagues to raise awareness for these important diseases. Our recent financial transactions, including the January 2021 follow-on common stock offering, provide us with necessary resources to continue to advance our lead programs and research pipeline. And lastly, just to summarize, this week we've begun the process to move into our new corporate headquarters. A lot going on so far in 2021, and with that, I'll turn the call over to Steve.