Good day, ladies and gentlemen, and thank you for standing by. Welcome to the REGENXBIO Inc. Third Quarter 2016 Earnings Conference Call. [Operator Instructions] As a reminder, this conference call is... [Audio Gap] I would now like to introduce your host for today's presentation, Mr. Patrick Christmas, Senior Vice President and General Counsel. Sir, please begin.

Good afternoon, and thank you for joining the call. With me 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 quarter ended September 30, 2016. 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 such words 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 section of REGENXBIO's annual report on Form 10-K for the fiscal year ended December 31, 2015, and quarterly report on Form 10-Q for the quarter ended September 30, 2016, which are 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, 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 is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially. I'll now turn the call over to Ken.

Thank you, Patrick, and good afternoon, everyone. Thank you for joining us today. On this call, we will provide you with an update on the company and our ongoing development programs, review accomplishments, progress made over the last 3 months, share financial results for the third quarter and look ahead to milestones for the remainder of the year. After that, we will open up the call for any questions. I'm going to start by reviewing our key areas of focus. We are developing our proprietary NAV Technology Platform with the vision of realizing potential of gene therapy to improve the lives of patients suffering from severe diseases. In order to accomplish this vision, we are focusing our development efforts on building our internal pipeline of lead product candidates while also licensing our NAV vectors to third-party companies in the gene therapy space who share our vision. As of September 30, 2016, our NAV Technology Platform was being applied in the development of 29 product candidates, including 5 internally developed product candidates and 24 candidates being developed by our NAV Technology licensees. We believe that our NAV Technology Platform, which is supported by the breadth and depth of our intellectual property portfolio, coupled with our focused internal and external development strategy, establish us as the leader in the AAV gene therapy space. As a reminder, our NAV Technology Platform is a proprietary AAV gene delivery platform that consists of more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. These AAV vectors have demonstrated clinical efficacy in severe genetic, central nervous system and hematological disorders and share the following important and differentiating attributes when compared to earlier-generation AAV vectors: higher and longer-term gene expression, broad and novel tissue selectivity, lower immune response and improved manufacturability. Our therapeutic focus at…

Thank you, Ken. REGENXBIO ended the quarter on September 30, 2016, with cash, cash equivalents and marketable securities totaling $184.9 million as compared to $216.4 million at December 31, 2015, a decrease of $31.5 million. REGENXBIO now expects full year 2016 cash burn to be between $55 million and $60 million, a decrease from our previous guidance of cash burn between $60 million and $70 million. Research and development expenses were $12.6 million for the quarter ended September 30, 2016, compared to $5.7 million during the same period in 2015. The increased R&D expenses in the 2016 period were primarily due to increased headcount of research and development personnel and increases in preclinical research and development, manufacturing and clinical trial expenses. General and administrative expenses were $6.2 million for the quarter ended September 30, 2016, compared to $2.6 million during the same period in 2015. The increased G&A expenses were primarily due to the growth of the G&A organization and establishing infrastructure to meet the requirements of a public company and our business objectives. REGENXBIO reported a net loss of $18.2 million or $0.69 per basic and diluted common share for the quarter ended September 30, 2016, compared to a net loss of $7.3 million or $1.52 per basic and diluted common share for the same period in 2015. As of September 30, 2016, we had 26.5 million common shares outstanding. With that, I'll turn the call back to Ken Mills, President and CEO of REGENXBIO, for a discussion of our expected 2016 milestones.

Thanks, Vit. So as we look to the remainder of 2016, we expect to meet our goal of enrolling our first patient in our Phase I/II clinical trial for RGX-501. We also continue to advance our pipeline of AAV gene therapies. As such, our preclinical studies for RGX-314 continue and we plan to file an IND for the treatment of wet AMD in the first quarter of 2017. Following that, we plan to file INDs for RGX-111 and RGX-121 in the first half of 2017. For the remainder of the year, we will be executing and advancing our clinical programs while optimizing our preclinical development strategies to best prepare us for success in the clinic. With strong science; a dedicated and talented team; a robust, diversified gene therapy program and solid financial position, I believe we're well on our way to becoming a leader in the gene therapy space. I want to extend my thanks to our strong investor base, our Board of Directors and the team here at REGENXBIO for your continued effort and support. With that, I'd like to open the call up to questions. Operator?

[Operator Instructions] Our first question or comment comes from the line of Matthew Harrison from Morgan Stanley.

This is Cyrus on for Matt. So a question about your cash spending guidance that went down for the year. So what was the impetus for this change? Is it a delay in some of your programs? Or are you guys just spending less overall?

Thank you for the question. As the year has progressed, we have achieved greater clarity into our expenses. As a result, we have realized that costs have come in lower across the whole company.

Great. And then a second question. For the HoFH trial, how are you guys going about screening? Is it faster with the protocol changes? And how quickly do you think you'll enroll patients beyond the first?

Yes, thanks, Cyrus. This is Ken. I'll field that one. So I think we have seen some meaningful improvement in the screening of the study since we made the protocol changes that we talked about at the end of the last quarter. And the focus has been very much recently on getting patients, who are now eligible for the study lined up in order that a schedule will allow us to fill out the dosing of the cohorts that we have in the study design as efficiently as possible. So it hasn't just been an effort to focus on first patient in, but it's actually been an effort to leverage the changes to the screening protocol, see improvements in that and set ourselves up for not only achieving the end-of-year goal but also come into 2017 with an expectation to complete the study as efficiently as possible.

Our next question or comment comes from the line of Ying Huang from Bank of America Merrill Lynch.

Firstly, maybe, Ken, I have a general question on the safety of gene therapy because we have seen some of the reactions from, for example, ATTC program and also Spark's hemophilia program in terms of immune reactions. Can you tell us whether you can avoid anything like that based on the preclinical data you have on your compounds? And then secondly, on the preclinical safety data you're required to submit to the FDA before the IND filing on RGX-314, I was just wondering if you have already submitted all the required data to FDA so that you have no other, I guess, issues that need to resolve before the IND.

Thanks for the questions. This is Ken. So I think that it's a great question. There definitely has been a lot going on this year in 2016 with respect to new reports of data about the AAV gene therapy platform in general. I know some but not all of what you're referring to with respect to different parties that have been reporting data. I don't know the details of some of the things you mentioned, but I would say that we definitely understand that there's a potential for there to be differences between the NAV Technology Platform and companies who aren't using the NAV Technology Platform. And that can be differences both in expression level, in durability and also in immunology. So it's hard to say anything more than that. With respect to how we view safety of REGENX programs, we think that the NAV Technology Platform conveys a lot of important benefits that have the potential to improve the profile, both from an efficacy, safety and manufacturability perspective. With respect to RGX-314, let me clarify if it wasn't apparent from the read-in where we are. So we're finalizing tox and biodistribution studies internal to the company right now, Ying. In fact, most of the experimental work is done, and so we've migrated to analysis and the beginning of report writing, which will still take several months. And that's why -- that's our target for filing of the IND in the first quarter of next year.

[Operator Instructions] I'm showing no additional audio questions. I'd like to turn the conference back over to management for any closing remarks.

Thanks very much for everyone participating today. We appreciate it, and we look forward to talking with you or seeing you again soon.

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