Good day, ladies and gentlemen, and welcome to the REGENXBIO Q1 2017 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference may be recorded. I would like to introduce your host for today's conference Mr. Patrick Christmas, Senior Vice President and General Counsel. You may begin.

Good afternoon, and thank you for joining us today. With us today are Ken Mills, REGENXBIO's President and Chief Executive Officer; and Vit Vasista, our Chief Financial Officer. Earlier this afternoon, REGENXBIO released financial and operating results for the three months ended March 31, 2017. The press release reporting our financial results is available on our website at www.regenxbio.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's discussion and analysis section of REGENXBIO's Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, which is 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 day of this call May 9, 2017, 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 maybe provided is preliminary, and does not purport to project financial positions or operating results of the Company. Actual results may differ materially. I would now like to turn the call over to Ken Mills, President and Chief Executive Officer of REGENXBIO. Ken?

Thank you, Patrick, and thank you all for joining us here this afternoon. On today's conference call, we'll share an overview of our progress over the past three months, an update on our development programs, and financial reports and results for the first quarter of 2017 as well as provide a look at our upcoming milestones. After that, we will open the call for questions. At REGENXBIO, our mission is to improve the lives through the curative potential of gene therapy based on our proprietary NAV Technology Platform. As a remainder, our AAV gene therapy product candidates are designed to deliver genes to cells to address genetic defects or enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our AAV gene therapy product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering important patient outcomes. We are currently advancing an internal pipeline of product candidates, which employ our proprietary NAV Technology Platform within three disease areas; retinal diseases, metabolic diseases and neurodegenerative diseases. These areas of focus have been strategically selected because we believe they can be uniquely addressable by our vectors [audio dip] there are many diseases with urgent unmet medical need. Beyond our internal development programs, we selectively license components of our NAV Technology Platform, most typically single vectors for single indications to third-party companies whose vision and commitment to gene therapy is aligned with ours. Our NAV Technology Platform consists of over 100 novel adeno-associated viral vectors or AAV vectors, including vectors like AAV7, AAV8, AAV9 and AAVrh10. Our vectors are the result of world-class research conducted at the University of Pennsylvania in the lab of Jim Wilson. These efforts were aimed at discovering and developing safer and more effective…

Thank you, Ken. REGENXBIO ended the quarter on March 31, 2017 with cash, cash equivalents and marketable securities totaling, $209.6 million as compared to $159 million as of December 31, 2016, an increase of $50.6 million. Cash, cash equivalents and marketable securities as of March 31, 2017 includes aggregate net proceeds of $70.8 million received from the previously announced underwritten public offering of common stock which closed in March 2017. Research and development expenses were $16.6 million for the three months ended March 31, 2017, compared to $6.2 million during the same period in 2016. This increase was primarily due to increased personnel costs and increases in preclinical research and development, manufacturing and clinical trial expenses. General and administrative expenses were $6.6 million for the three months ended March 31, 2017, compared to $5.5 million during the same period in 2016. This increase was primarily due to the continued establishment of the infrastructure necessary to meet our business objectives. The net loss was $22 million or $0.82 per basic and diluted common share for the three months ended March 31, 2017, compared to a net loss of $10.8 million or $0.41 per basic and diluted common share, for the three months ended March 31, 2016. As of May 5, 2017, we had 30.8 million common shares outstanding. REGENXBIO reiterates that it expects full-year 2017 cash burn to be between $75 million and $85 million. Full-year 2017 cash burn excludes the effect of REGENXBIO's previously announced underwritten public offering of common stock in March 2017 and the underwriters' exercise of their option to purchase additional shares in 2017, which resulted in aggregate net proceeds through REGENXBIO of approximately $81.5 million after deducting underwriting discounts and commissions and estimated offering expenses. With that, I will turn the call back to Ken to review our upcoming 2017 milestones.

Thank you, Vit. We continue to look forward to ongoing clinical advancements of both our internal and partnered programs. With the March 2017 initiation of the Phase I trial for RGX-501 for HoFH and with the Phase I trial for RGX-314 for wet AMD on track for enrollment by mid-2017. We expect to report our first interim trial updates in each study by the end of this year. Additionally, we plan to file the INDs for RGX-111 and RGX-121 in mid-2017. With our recently augmented cash position and strengthened management team, we look forward to executing on these upcoming milestones and providing you with further updates on patient enrolment in clinical data this year. I like to take this time to especially acknowledge and thank the individuals and families who volunteered to take part in our clinical trials. Their participation is crucial to advancing research and development of our novel therapies that may dramatically improve lives and we're grateful to them. With that, I'd like to open up the call for any questions. Operator?

Thank you. [Operator instructions] And our first question comes from the line of Matthew Harrison from Morgan Stanley. Your line is now open.

Matthew, are you there?

Yes. Hi. Sorry about that. This is Vikram on for Matthew. So we had a question about what kind of data we could expect from the interim update for 501 and 314, specifically for 314 there's often been mixed results and different kinds of endpoints reported for wet AMD programs. Any insight you could provide about what kind of data that could be presented would be helpful?

Sure Vikram, and thanks for the question. So I think when we initiate the study, we will have an outline of the progress of enrollments over the course of the year. And by the time we get to the end of 2017, we expect that we will have made meaningful progress in that kind of dose escalation study design, probably having focused on working through at least the first two dose cohorts of patients and perhaps even expecting to have initiated dosing of the third dose cohort. For each of the cohorts when they receive RGX-314 for follow-up of up to six months, we will be making measures of visual acuity looking at imaging using spectral OCT that I described, as well as taking measures of fluid from the aqueous of eyes that have been injected with RGX-314 in order to also measure a protein product that we expect will be expressed from the delivery of the RGX-314 treatment. So among those outcome measures at different time points depending on when patients have been dosed, that's all constitute basically the package of the interim update at the end of the year.

Okay. That's helpful. Thank you. End of Q&A

Thank you. [Operator Instructions] And I'm not showing any further questions over the phone line at this time. I'd like to turn the call back over to Ken Mills, REGENXBIO's President and CEO, for closing remarks.

Thank you very much, and thanks to everyone for joining us on the call this afternoon. We look forward to advancing our pipeline through the rest of this year and providing further updates on our progress. Have a great afternoon.

Ladies and gentlemen, thank you for your participation in today's conference. This concludes the program and you may now disconnect. Everyone have a great day.