REGENXBIO Inc.

and Vit Vasista, our Chief Financial Officer. Earlier this afternoon, Regenexx Bio released financial and operating results for the third quarter ended September 30, 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's discussion and analysis sections of Regenexx BIO's annual report on Form 10-K for the full year ended December 31st, 2020, and comparable risk factors 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 the date of this call, November 2, 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. Actual results may differ materially. I would now like to turn the call over to Ken Mills. Ken?

Ken Mills Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I'm pleased to begin today's call with a recap of our recent business highlights, including the strategic eye care collaboration with AbbVie for the development and commercialization of RGX314. Steve will then provide an update on our clinical programs, and Vit will provide an overview of financial results for the third quarter of 2021. At the end of the call, we will open up the line for questions. To start, I'm very excited about our recent news of our partnership with AbbVie to develop and commercialize RGX314, our one-time gene therapy for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic eye disorders. We believe AbbVie is a strong and complementary partner for Regenexx Bio, and we plan to leverage their commercial infrastructure and leadership in eye care with our expertise in AAV gene therapy clinical development and our deep in-house knowledge of manufacturing and production. We believe this is an important partnership that could expand the impact of RGX314 for millions of patients around the world. The transaction is expected to close by the end of 2021, subject to the satisfaction of customary closing conditions, including applicable regulatory approvals. Now, Steve will shortly review our recently announced positive initial data from our ongoing Phase II trials of RGX314 for the treatment of wet AMD and diabetic retinopathy using in-office suprachoroidal delivery. We're pleased with the early clinical profile around suprachoroidal delivery. Looking ahead, we plan to report additional interim data from our Phase II trial in wet AMD using suprachoroidal delivery at the American Academy of Ophthalmology 2021 annual meeting later this month. We also continue our development of innovative gene therapy treatments beyond ophthalmology, including our CNS platform programs, RGX121 and RGX111, for the treatment of Hunter syndrome and Hurler syndrome, respectively, as well as RGX202 for the treatment of Duchenne muscular dystrophy. I want to take this moment to thank our Regenexx bio team, our clinical investigators, and the patient communities for their steadfast commitment to the development of innovative gene therapies. As we look forward to 2022, we believe we're well-positioned to drive the development of potentially curative one-time gene therapies for patients And with that, I'm going to turn the call over to Steve for a bit more detail on the clinical and regulatory status of our program.

Thanks, Ken. We continue to enroll patients in the atmosphere. The first of two pivotal plan trials to evaluate efficacy and safety of the subretinal delivery of RGX314 for the treatment of wet AMD. We look forward to continued execution across this pivotal program, including the initiation of our second pivotal trial, which we expect to take place later this year. We're encouraged by the emerging clinical profile of RGX314 from the Phase II ABA trial for the treatment of wet AMD, which was announced at the Retina Society meeting at the This was the first ever data reported from a gene therapy delivered to the supracoroidal space of the eye in a clinical trial. At six months after one-time administration of RGF314, patients at the first dose level were observed to have stable visual acuity and retinal thickness, along with a 75.9% reduction in anti-VEGF treatment burden. compared to the mean annualized injection rate during the 12 months prior to RGF314 administration. Looking at the safety profile, RGF314 was reported to be well-tolerated across 50 patients dosed in cohorts 1 through 3 as of September 13, 2021. Among patients in cohort 1, common treatment emergent adverse events in the study eye were generally mild and none were severe. Mild intraocular inflammation was observed in 4 out of 15 patients, and all cases were resolved within days to weeks on topical corticosteroids, which have been discontinued. These cases of inflammation were asymptomatic and observed on slit-lamp examination, and it's important to remember that these patients were not receiving prophylactic steroids before or after administration of RGF report change. We also announced the expansion of the ABA trial to enroll patients in two additional cohorts to evaluate a third dose level of 1E12 genome copies per eye. Cohort four will enroll 15 patients who will receive RGX314 at this dose level, and cohort five will evaluate RGX314 at the same dose level as cohort four in 20 patients who are neutralizing antibody positive. As Ken mentioned, our next update from this trial, which will include six-month data from cohort two, will be presented at the upcoming AAO conference in mid-November. We also presented positive initial data from our altitude trial, which is our phase two trial to evaluate RGX314 for the treatment of diabetic retinopathy, or DR, using superchoroidal delivery at the ASRS conference in mid-October. DR can start in young adulthood and often progresses quickly, leading to vision-threatening complications. including diabetic macular edema, or DME, and neovascularization that can lead to vision loss. It's estimated that one-third of DR patients in the United States have moderate to severe NPDR without DME, equal to about 3 million patients. There are treatment options for patients with DR, including chronic repeated anti-VEGF injections, retinal laser treatment, and surgery. However, most patients with NPDR go untreated, as the current standard of care is watchful waiting until vision becomes threatened. Unfortunately, without treatment, a large proportion of these patients will eventually develop vision-threatening complications, including diabetic macular edema and neovascularization that can lead to blindness. We believe that one-time treatment with RGF314 has the potential to provide sustainable long-term anti-VEGF protein production in the eye, which could reduce the severity of DR and prevent vision-threatening complications. In the altitude trial as of September 29th, RGF314 was reported to be well-tolerated in the 15 patients dosed in cohort 1, and no intraocular inflammation was observed on slit lamp examination. Thirty-three percent of patients dosed with RGF314 in cohort 1 demonstrated a two-step or greater improvement from baseline on the diabetic retinopathy severity scale, or DRSS, at three months earlier. compared to zero of the five patients of the observational control arm. Of note, one RGX314 treated patient had a four-step improvement. We're encouraged to see these results at this early time point of three months after the one-time administration. In the altitude trial, dosing of patients at the second dose level in cohorts two and three is ongoing, and we look forward to providing additional updates from this trial next year. We believe the preliminary safety profile from our AV8 and altitude trials support continued development of the supracoroidal route of delivery of RGX314. Shifting focus to our rare genetic disease programs, our team is on track to submit an investigational new drug application, or IND, to the FDA for RGX202, a potential one-time year-end 2021. From our ongoing Phase 1-2 trial of RGX121 in MPS II patients up to five years old, we continue to enroll patients and today announce that we have expanded Cohort 3 to include up to six additional patients. We expect to report additional data from this trial in the first half of 2022. Enrollment is ongoing in cohort two of the Phase 1-2 trial of RGX111 for the treatment of MPS1, and we expect to share initial data from this trial in the first half of 2022. We continue to evaluate the path forward for the RGX181 program for the treatment of CLM2 disease, and we plan to provide an update in 2022. We are also conducting additional preclinical studies of RGF381 for the treatment of ocular manifestations of CLN2 disease, and we are in discussions with regulatory agencies. We plan to provide a program update in 2022. We have made meaningful progress across our entire portfolio of gene therapy candidates throughout 2021, and we look forward to building on this momentum for the remainder of this year and into 2022. With that, I turn the call back over to Ken.