REGENXBIO Inc.

Welcome everyone to the Regenexx BIO RGX 202 Topline Pivotal Data Webcast. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of Regenexx BIO. Please go ahead.

Good morning, and thank you for joining us today. Earlier this morning, Regenexx Bio released pivotal, top-line data from the Affinity Duchenne trial of RGX202, as well as financial and operating results for the first quarter ended March 31, 2026. The press releases are available on our website at www.regenexxbio.com. Today's webcast 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 sections of Regenexx Bio's annual report on Form 10-K for the full year ended December 31st, 2025, and comparable risk factor sections of Regenexx Bio's quarterly reports on Form 10-Q, 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, May 14th, 2026, 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. Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of Regenexx Bio. Curran?

Thank you, Patrick, and good morning, everyone. Thank you for joining us. Earlier this morning, we announced positive topline data from the pivotal trial of RGX202, our potential best-in-class investigational gene therapy for Duchenne muscular dystrophy. This data is highly meaningful for us and for patients, and we are excited to share it with you today. We have shared very encouraging Phase 1-2 results for RGX202 previously, and today I'm very pleased to have a group of experts with us to reflect on the first Phase 3 results from our Affinity Duchenne trial. Our chief medical officer, Dr. Steve Bacola, will present the data before opening the call to Dr. Aravindan Virapandian, also known by his patients as Dr. Panda, Dr. Carolina Tessierosha, and Dr. Diana Castro to share their perspectives. We're also very pleased that Dr. Panda will share videos from his patients treated with RGX202 to give you a sense of what this positive data looks like for families in a real world setting. Before jumping into the data, I'll provide a quick recap of the first quarter 2026 earnings, which we also reported this morning. Regenexx Bio continues executing against our mission to deliver multiple first and best in class gene therapies. As you'll hear today, the positive data and momentum continue for RGX202. Across the pivotal and confirmatory studies, we have dosed more than 50 patients with line of sight to dosing 60 patients by mid-year. Supported by today's updates, we are planning for a potential approval in 2027. In retinal disease, we are on track to dose the first patient in the phase 2B trial for diabetic retinopathy in the second quarter, which would provide $100 million milestone payment from our partner, AbbVie. Additionally, preparations continue to report top line data from the subretinal pivotal studies in the fourth quarter. I'm also very pleased to share the partial clinical hold placed on RGX 121 for the treatment of Hunter syndrome has been fully lifted. We recently filed an appeal of the 121 CRL and are continuing to engage the agency regarding a path forward for the program. Overall, I am happy to share that we remain well positioned to have three approvals over the next couple of years, including two blockbuster opportunities. Turning back to our main event, the RGX202 Pivotal Top Line Results. Today's positive data update is especially meaningful as we look at the impact of this progressive devastating disease. High unmet need remains for Duchenne patients as current options face limitations related to efficacy, safety, and access. The untreated Duchenne population continues to grow in the US and globally. Physicians and patients need new next generation options. Our Duchenne gene therapy program is differentiated from other gene therapies in multiple ways. Our novel construct with the C-terminal domain enables us to deliver a microdystrophin with more key elements of the full-length dystrophin that's missing in boys with Duchenne. With the CT domain, RGX202 is uniquely designed to better preserve and protect the muscle, as demonstrated in preclinical studies. This novel construct combined with our proactive short course immune suppression regimen and leading product purity levels allows us to maximize the potential for therapeutic benefit while maintaining an impressive safety profile. I'll now turn the call over to Steve to walk us through the exciting data that reinforces our confidence in RGX202 as a potential best in class therapy for patients.