REGENXBIO Inc.

on our website at www.regenixbio.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 sections of Regenics Bio's annual report on Form 10-K for the full year ended December 31, 2022, and comparable risk factors section of Regenics 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, August 2nd, 2023, 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 report 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, CEO of Regenexx Bio. Ken?

Thank you, Patrick. Good afternoon, everyone. Thanks for joining us. I'm pleased to begin today's call with a recap of some recent business highlights, as well as an update on our corporate goals. Dr. Steve Pecola, our Chief Medical Officer, will then provide an update on our clinical programs, and Vic Pesista, our Chief Financial Officer, will provide an overview of financial results for the second quarter ended June 30th, 2023. At the end of the call, we will open up the line for questions. At Regenexx Bio, our mission is to improve lives through the curative potential of gene therapy, and we're focused on developing therapies for diseases that have significant unmet needs. We continue to be a leader in gene therapy, with thousands of patients who have been dosed with AAD therapeutics derived from our proprietary NAV technology platform. Recently, it's been a significant time of growth for the AAD gene therapy industry and at Regenexx Bio. We're developing a deeper understanding of gene therapy market access models and digesting regulatory approvals, including the FDA's support of biomarkers as surrogate endpoints to support accelerated approvals. There are now five FDA-approved AAV therapeutics. And I'm pleased how Regenexx Bio remains a leader in the gene therapy space, as our five-by-25 strategy is on track for advancing five AAV therapeutics from our internal pipeline and licensed programs into pivotal stage or commercial products by 2025. What distinguishes Regenexx Bio as a leader in AAV therapeutics? It's our platform, pipeline, and products. There are thousands of patients who have been dosed with AAV Therapeutics derived from our NAV technology platform and hundreds more are receiving treatment every quarter. We estimate approximately two children per day are receiving Zolgensma, which uses our NAV AAV9 vector. We believe that we have a strong pipeline of AAV Therapeutics ourselves with the potential to deliver one-time treatments to address significant unmet need for patients living with common and rare diseases. At Regenexx Bio, we are conducting over 12 active clinical trials using six different forms of delivery devices in three therapeutic areas, retinal, neuromuscular, and neurodegenerative diseases. In 2023, our team is executing on two pivotal phase programs that include over 1,000 subjects to support a goal we have to file our first BLAs in 2024 and 2025. We're observing that, on average, one new patient is being dosed in Regenexx bioclinical trials daily. Our work in retinal disease primarily centers around the strategic partnership we entered into with AbbVie at the end of 2021 to develop and commercialize ABBV-RGX314, or 314, and investigational gene therapy. AbbVie is a strong and complementary partner for Regenexx Bio. We expect to leverage AbbVie's global development and commercial infrastructure within eye care with our expertise in AAV gene therapy clinical development and deep in-house knowledge of manufacturing. Together, we're developing 314 to be the first one-time therapeutic option in major retinal vascular diseases to address significant unmet need for patients. Overall, our pipeline of AEB therapeutic candidates is addressing high unmet need for millions of patients in diseases that represent current market opportunities of over $20 billion. Just a few weeks ago, at our investor day in July, we introduced some new clinical trial data from our retina programs using suprachoroidal delivery and our Duchenne microdystrophin candidate, and we provided specific guidance about additional upcoming interim trial updates From our aviate and altitude trials, we reported that mild to moderate intraocular inflammation previously observed with suprachoroidal delivery is mitigated with short-course topical steroid eye drops. And we reported safety updates from the initial dosings in cohort one of the Affinity Duchenne study to support a well-tolerated profile of our candidate RGX202 to date. Now I'll turn the call over to Steve so that he can review in detail a bit more about the clinical progress and pipeline updates.

Thanks, Ken. I'll begin with 314, which is being developed in collaboration with AbbVie to treat wet AMD and diabetic retinopathy via subretinal and suprachoroidal routes of administration. 314 utilizes our NAVAVA vector to deliver a gene encoding, a therapeutic antibody fragment to inhibit VEGF. The anti-VEGF market opportunity is poised to grow significantly as the population ages. 314 for the treatment of wet AMD via subretinal delivery is being evaluated in two ongoing pivotal trials, Atmosphere and Ascent. We recently announced the expansion of these studies to enroll a total of 1,200 patients in the US, Europe, Japan, and Israel to support global development of the program. We also recently initiated a fellow eye treatment study as part of the pivotal program using subretinal delivery. This study is evaluating the safety, efficacy, and immunogenicity of subretinal 314 administration in the fellow eye of patients with bilateral disease from atmosphere and ascent who previously received a subretinal injection of 314. Overall, we plan to complete all these trials in time to support global regulatory submissions in late 2025 through the first half of 2026. Additionally, earlier this week, we presented new interim results from our phase two pharmacodynamics study designed to evaluate the same dose levels being used in the two pivotal trials. The updated interim data demonstrated that 314 manufactured using our NavXRest platform process was well-tolerated, and in both the low-dose and high-dose cohorts through six months, patients achieved expected protein levels, along with stable to improved BCVA and CRT, as well as meaningful reductions in anti-VEGF burden, with most subjects remaining injection-free. This study is now fully enrolled. We also have two ongoing phase two trials that fall under our collaboration with AbbVie, assessing in-office supracoroidal delivery of 314 for treatment of wet AMD in the AVIATE trial and treatment of diabetic retinopathy in the ALTITUDE study. AVIATE is an active control dose escalation trial evaluating 314 for the treatment of wet AMD. We recently presented safety data at our investor day from cohort six evaluating dose level 3, 1E12, GC per eye, that included short-course prophylactic ocular steroids following administration of 314. The initial data presented continues to support the safety profile of 314 and highlighted the inclusion of short-course prophylactic steroid eye drops, which resulted in zero cases of intraocular inflammation, or IOI, in all patients. We plan to present full six month results from cohorts five and six at the Hawaii and I meeting in the beginning of 2024. Altitude is the active controlled dose escalation study of 314, suprachoroidal delivery for treatment of DR. We're very excited about the opportunity in DR given the size of the market which exceeds that of wet AMD and because we believe this patient population can benefit the most from a potential one-time gene therapy. During our investor day, we presented initial interim data from cohorts four and five at dose level three with short course prophylactic steroid eye drops following 314 administration. The data demonstrated that 314 was well tolerated with no drug-related serious adverse events in 29 patients from these cohorts. And just as observed in wet AMD, the inclusion of short-course prophylactic steroid eye drops resulted in zero cases of IOI in all patients. We look forward to presenting full 12-month results from cohorts one to three at the American Academy of Ophthalmology meeting later this year. Moving to Duchenne, as Ken mentioned in his remarks, At our investor day, we were pleased to announce our new exon skipping program to complement RGX202. Duchenne patients face high unmet need, and we are committed to bringing multiple treatment options for these boys. Our first program, RGX202, is a potential one-time gene therapy for the treatment of Duchenne, being developed as a highly differentiated product designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the CT domain found naturally in occurring dystrophin. RGX202 is designed to support the delivery and targeted gene expression throughout skeletal and heart muscle using our NAVAV8 vector and a well-characterized muscle-specific promoter. During our investor day, we reported safety data from the Phase 1-2 Affinity Duchenne trial The data we presented on the two patients, ages 4 and 10, showed that RGX202 was well-tolerated in both patients with no drug-related serious adverse events. Time of post-administration follow-up was 45 days and more than three months. We continue to actively recruit patients in this trial, and we look forward to presenting additional data at the World Muscle Society Congress later this year that will include longer-term safety data and initial microdystrophin protein expression levels in muscle at three months. We also continue to enroll patients in Affinity Beyond, an observational screening study that is evaluating the prevalence of AAV8 antibodies in patients with Duchenne. Moving to our other rare disease programs, we are developing two programs for mucopolysaccharide OCs, MPS-2 and MPS-1. RGX-121 is an investigational one-time AAV therapeutic for the treatment of MPS-2, also known as Hunter syndrome, being evaluated in the ongoing Phase 1-2-3 campsite trial. In May, we announced that we received regenerative medicine, advanced therapy, or RMAT designation from the FDA. Recognizing the preliminary data we have presented to date indicates its potential to address the unmet medical need for patients with Hunter syndrome. We completed enrollment of 10 patients for our campsite trial in the first half of 2023 and remain on track to support a BLA filing in 2024 using the accelerated approval pathway. Now on to RGX111, an investigation on one-time AAV therapeutic for the treatment of severe MPS1. We have completed enrollment of the Phase 1-2 trial, and we remain on track to share additional updates on plans for this program later this year. In addition to these two programs, we also are developing RGX181 to treat the neurodegenerative manifestations and RGX381 to treat the ocular manifestations of CLM2 or Batten disease. Physician investigators in Brazil continue with follow-up for the first child with CLN2 disease dosed with RGX181 under a single-patient investigator-initiated study, and we expect investigators to report initial interim data from the single patient, including six-month results at the Society for the Study of Inborn Errors of Metabolism annual symposium later this year. We are also happy to report the recent dosing of our first patient with RGX381. To conclude, we have made significant progress with data updates and trial advancements across all our programs as we continue toward our goal of 5 by 25. Lastly, I'd like to thank the patients, families, clinicians, and patient advocacy representatives who are involved in and support all these trials. And with that, I turn the call over to Vit to review our financial guidance.