Retinal Artery Occlusion

iStock, Love Employee 4D Molecular Therapeutics reported its experimental gene therapy demonstrated a nearly 90% reduction in the need for annualized standard-of-care injections in patients with wet age-related macular degeneration. 4D Molecular Therapeutics announced on Wednesday that its investigational gene therapy 4D-150 demonstrated durable clinical activity in the Phase I/II PRISM study of patients with wet age-related macular degeneration. In a 30-patient cohort, 4D-150 cut the need for standard-of-care injections by 89% through 52 weeks of follow-up, and 70% of these patients were injection-free, according to 4DMT’s announcement. In another cohort of 27 patients with severe disease, the need for standard-of-care treatment dropped by 83% and 44% were injection-free. The gene therapy also resulted in sustained benefits in terms of central subfield thickness and mean best-corrected visual acuity. In terms of safety, PRISM found 4D-150 to be well-tolerated. The frequency for of intraocular inflammation (IOI) was consistent with approved anti-VEGF therapies, which are currently widely used to treat age-related macular degeneration (AMD). Overall, 4DMT documented two IOI episodes in all 71 wet AMD patients enrolled, yielding an incidence rate of 2.8%. There were no cases of retinal artery occlusions, hypotony, vasculitis, choroidal effusions and endophthalmitis related to the gene therapy. 4DMT CEO David Kirn in a statement called 4D-150 a “potentially paradigm shifting product candidate,” noting that PRISM’s findings highlight “clear reduction in overall treatment burden and potential multiyear clinical benefit in previously treated patients.” The California-based biotech will continue to invest in 4D-150’s development, Kirn said, and push it into late-stage studies. Alongside the PRISM readout on Wednesday, 4DMT detailed the design of its Phase III 4FRONT development program for 4D-150, which is set to include two double-masked, randomized non-inferiority trials, compared with on-label treatment with Bayer and Regeneron’s Eylea (aflibercept). The gene therapy will be given at a single 3E10 vg/eye dose. The first study under this program, dubbed 4FRONT-1, has a target enrollment of 500 patients and is set to start in the first quarter of 2025, Kirn said. The Phase III program design is in alignment with feedback from the FDA under its Regenerative Medicine Advanced Therapy Designation, according to 4DMT. 4D-150 is an investigational gene therapy that uses an intravitreal vector to deliver a transgene that encodes for aflibercept—the active ingredient of Eylea—and an RNAi molecule that blocks the VEGF-C protein. This mechanism of action allows 4D-150 to suppress four different VEGF proteins in the eye, disrupting the key disease pathway in wet AMD. With 4D-150, 4DMT is seeking to offer a one-time alternative to Eylea, which is injected into the eye every eight weeks , following an initiation regimen. Eylea has become a top-selling treatment in the ophthalmology space. In 2023, the product brought in $5.9 billion in sales worldwide.

4D-150 demonstrated robust and durable clinical activity across all wet age-related macular degeneration (wet AMD) patient populations based on longest available follow-up data In broad population (Phase 2b), 70% injection-free through 52 weeksIn severe population (Phase 1/2a), 83% overall reduction in annualized injections through 52 weeks 4D-150 continues to be safe and well tolerated with intraocular inflammation (IOI) profile numerically similar (2.8% transient 1+ vitreous cells) to approved anti-VEGF agents4FRONT Phase 3 program designed to maximize 4D-150’s potential, including probabilities of clinical, regulatory and commercial success across global marketsCorporate webcast to be held on September 18, 2024 at 4:15 p.m. ET EMERYVILLE, Calif., Sept. 18, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced data showing continued robust and durable clinical activity, based on longest interim follow-up data from the Phase 1/2 PRISM clinical trial, and 4FRONT Phase 3 study design, which will be presented at its 4D-150 Wet AMD Development Day. “We continue to build support for 4D-150 with positive interim data from PRISM showcasing clear reduction in overall treatment burden and potential multiyear clinical benefit in previously treated patients, with an emerging safety profile comparable to approved anti-VEGF agents. In addition, we have assembled an exceptional senior leadership team with deep late-stage drug development, regulatory and commercial experience in large market ophthalmology to design and execute our upcoming pivotal studies,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “We expect to initiate 4FRONT-1, our first 4D-150 Phase 3 study in wet AMD, in Q1 2025. We look forward to continuing the rapid advancement of this potentially paradigm shifting product candidate that addresses the limits of current treatment options for patients with wet AMD.” “As prudent medicine developers, we began our clinical development program for 4D-150 in the most severe wet AMD patients in the Phase 1/2a portion of PRISM. After observing favorable tolerability and clinical activity results, we and our investigators felt confident in 4D-150’s potential across a broad range of wet AMD patients and designed the Phase 2b cohort,” said Robert Kim, M.D., Chief Medical Officer of 4DMT. “We believe the interim data from Phase 2b demonstrates strong clinical activity, especially in more recently diagnosed patients.” 4D-150 Wet AMD Development Day Key Highlights: Positive Interim Data from 4D-150 Phase 1/2 PRISM Study Clinical Activity (based on data cutoff of September 3, 2024): Robust and durable treatment burden reduction observed in all PRISM populations studied with the planned Phase 3 dose of 3E10 vg/eye of 4D-150 (*Based on Kaplan-Meier method for calculating endpoint with follow-up through 52 weeks (Phase 1/2a) and variable follow-up through 32–52 weeks (Phase 2b); **Defined as diagnosed ≤6 months) Phase 1/2a Severe (n=24, through 52 weeks): 83% overall reduction in annualized injections52% received 0 or 1 injection*44% injection-free* Phase 2b Broad (n=30, through 52 weeks): 89% overall reduction in annualized injections80% received 0 or 1 injection*70% injection-free* Phase 2b Recently Diagnosed** (n=15, through 52 weeks): 98% overall reduction in annualized injections100% received 0 or 1 injection*87% injection-free* Central Subfield Thickness (CST): sustained anatomic control with fewer fluctuationsMean best corrected visual acuity (BCVA): stable (Phase 1/2a) or sustained improved (Phase 2b) Safety (based on data cutoff of August 23, 2024): 4D-150 continues to be well tolerated with favorable safety profileRate of 4D-150 IOI numerically similar to that reported for approved anti-VEGF agents Wet AMD: 2.8% (2 of 71) had 4D-150–related IOI at any timepoint 2 patients had transient 1+ vitreous cells 99% (70 of 71) completed steroid prophylaxis taper on schedule97% (69 of 71) remained off steroids completely Diabetic Macular Edema (DME; SPECTRA trial): No patients treated at any dose (n=22) have experienced IOI events at any timepoint No 4D-150–related hypotony, endophthalmitis, vasculitis, choroidal effusions or retinal artery occlusions observed to date across both the wet AMD and the DME programs 4FRONT Wet AMD Planned Phase 3 Program Key Design Elements Company planning for global 4FRONT Phase 3 development program comparing a single dose of 4D-150 3E10 vg/eye to on-label aflibercept 2mg Q8 weeks: Initiation of 4FRONT-1 clinical trial (N=500) expected in Q1 2025Eligibility criteria: 1) Patients must be both recently diagnosed and treatment naïve wet AMD patients, and 2) Randomization requires on study demonstration of aflibercept responsiveness Program expected to include two double-masked, randomized, controlled noninferiority trials: Primary endpoint: noninferiority in BCVASham controlled to support maskingAll patients randomized to receive 3 total loading doses per aflibercept label Supplemental aflibercept injection criteria for 4D-150 arm optimized to protect primary BCVA endpoint and to maximize reduction of supplemental treatment burden; no supplemental injections allowed in control armStudy design has been aligned with feedback from U.S. Food and Drug Administration (FDA) under RMAT designationAlignment ongoing with European Medicines Agency (EMA) under PRIME designation Carlos Quezada-Ruiz, M.D., FASRS, SVP, Therapeutic Area Head, Ophthalmology of 4DMT added, “Given the consistent emerging safety profile and strong signs of promising clinical activity across a broad range of wet AMD patients, including three patients from our Phase 1a who have gone 2-3 years without the need for supplemental aflibercept injections, we are excited to rapidly advance 4D-150 into Phase 3. We have worked closely with global regulatory agencies and our Ophthalmology Advisory Board to maximize the probabilities of clinical, regulatory and commercial success of the 4FRONT Phase 3 program. In partnership with the retina community, we are eager to begin enrollment in 4FRONT-1 and potentially bring a paradigm shifting treatment option to patients.” “Our patients with wet AMD require frequent life-long treatment with intravitreal injections, leading to a high treatment burden and suboptimal outcomes in the real world compared to clinical trials,” said Arshad M. Khanani, M.D., M.A., FASRS, Director of Clinical Research at Sierra Eye Associates and Clinical Professor at University of Nevada, Reno. “Based on the data to date, 4D-150 has the potential to decrease treatment burden and control wet AMD with a safe, single routine intravitreal injection while maintaining vision and anatomy. I am looking forward to working with the 4DMT team, the Ophthalmology Advisory Board, and investigators on the 4FRONT Phase 3 global development program to advance this potential treatment option for all our patients with wet AMD.” 4D-150 Wet AMD Development Day Webcast Details Title:4D-150 Wet AMD Development DayDate/Time:Wednesday, September 18, 2024 from 4:15 p.m. to 6:15 p.m. ETRegistration:Link An archived copy of the webcast will be available for up to one year by visiting the “Investors & Media” section of the 4DMT website: https://ir.4dmoleculartherapeutics.com/events. About Wet AMD Wet AMD is a highly prevalent disease with estimated incidence rate of 200,000 new patients per year in the United States. It is estimated that the total prevalence of wet AMD in certain major markets, including the United States and the European Union (major markets), and Japan, will be greater than 4 million individuals in the next five years. Wet AMD is a type of macular degeneration where abnormal blood vessels (macular neovascularization or MNV) grow into the macula, the central area of the retina. As a consequence, MNV causes swelling and edema of the retina, bleeding and scarring, and causes visual distortion and reduced visual acuity. The proliferation and leakage of abnormal blood vessels is stimulated by VEGF. This process distorts and can potentially destroy central vision and may progress to blindness without treatment. About 4D-150 for Wet AMD 4D-150 combines our customized and evolved intravitreal vector, R100, and a transgene cassette that expresses both aflibercept and a VEGF-C inhibitory RNAi. This dual-transgene payload inhibits four members of the VEGF angiogenic family of factors that drive wet AMD and DME: VEGF A, B, C and PlGF. R100 was invented at 4DMT through our proprietary Therapeutic Vector Evolution platform; we developed this platform utilizing principles of directed evolution, a Nobel Prize-winning technology. 4D-150 is designed for single, low-dose intravitreal delivery for transgene expression from the retina without significant inflammation. About 4DMT 4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing six clinical-stage and one preclinical product candidate, each tailored to address rare and large market diseases in ophthalmology, pulmonology and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT. All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied. Learn more at www.4DMT.com and follow us on LinkedIn. Forward Looking Statements: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, clinical benefits of and market potential of 4DMT’s product candidates, as well as the plans, announcements and related timing for the clinical development of and regulatory interactions regarding 4D-150. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Contacts: Media: Katherine SmithInizio Evoke CommsMedia@4DMT.com Investors: Julian PeiHead of Investor Relations and Corporate FinanceInvestor.Relations@4DMT.com

Company remains on track for its first BLA filing in 2024 and is accelerating progress toward pivotal trial initiation for Duchenne (H2 2024) and diabetic retinopathy (H1 2025) New, positive data from Phase II AFFINITY DUCHENNE® trial of RGX-202 demonstrates consistent high expression of microdystrophin across treated patients in all age groups Successful End-of-Phase II meeting with FDA for RGX-202 supports plans for using the accelerated approval pathway and pivotal initiation in Q4 2024 End-of-Phase II meeting for ABBV-RGX-314 for diabetic retinopathy accelerated to Q4 2024 to support global pivotal program initiation in H1 2025 $327 million in cash, cash equivalents and marketable securities as of June 30, 2024, expected to fund operational runway into 2026 Conference call Thursday, August 1, at 4:30 p.m. ET ROCKVILLE, Md., Aug. 1, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the second quarter ending June 30, 2024. Recent operational highlights, including acceleration of the late-stage pipeline, support meaningful value generation from the Company's leading portfolio of AAV Therapeutics. "In the first half of 2024, REGENXBIO has made remarkable progress in accelerating and advancing the development of AAV Therapeutics that are well-positioned to make a profound impact for patients," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "Looking ahead, we have multiple, exciting catalysts across our pipeline, including enrolling our first patient aged 1-3 in the AFFINITY DUCHENNE® trial and advancing RGX-202 into pivotal stage to address the significant ongoing unmet need in this community, initiating a rolling BLA for RGX-121 as the only one-time treatment for Hunter syndrome, and accelerating our End-of-Phase II meeting with the FDA for ABBV-RGX-314 in diabetic retinopathy. Each of these programs represent differentiated therapies that we expect will drive significant value." PROGRAM HIGHLIGHTS AND MILESTONES Neuromuscular Disease: RGX-202 is an investigational one-time AAV Therapeutic designed to deliver a novel microdystrophin gene for improved function and outcomes in Duchenne. REGENXBIO recently announced new, positive data from two patients treated with dose level 2 (DL2), demonstrating consistent, high microdystrophin expression across treated patients in the Phase II AFFINITY DUCHENNE® trial of RGX-202. In patients aged 5.8 and 8.5 at dosing, RGX-202 microdystrophin expression was measured to be 77.2%, and 46.5%, respectively, compared to control at three months. As of July 8, 2024, RGX-202 has been well tolerated with no serious adverse events. All seven patients who completed three-month trial assessments indicate meaningful increases in expression of RGX-202 microdystrophin and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement. REGENXBIO expects to complete enrollment in the DL2 expansion cohort in early Q3 2024 and has initiated enrollment in the cohort for boys aged 1-3. The Company remains on track to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024. REGENXBIO recently held a successful End-of-Phase II (EOP2) meeting with the FDA and is moving forward with plans to initiate a pivotal trial in Q4 2024. Discussions with the FDA continue to support use of microdystrophin as a surrogate endpoint reasonably likely to predict clinical benefit for accelerated approval. The Company expects to share the pivotal trial design in late Q3 to early Q4 2024. RGX-202 is manufactured using REGENXBIO's proprietary, high-yielding NAVXpress™ platform process. This suspension-based manufacturing process has demonstrated scalability up to 2,000L with consistent yield and product purity. The REGENXBIO Manufacturing Innovation Center has the capacity and yields to produce up to 2,500 doses of RGX-202 per year to support future commercialization. Retinal Disease: ABBV-RGX‑314, being developed in collaboration with AbbVie, is a potential one-time treatment for chronic retinal conditions, including wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and diabetic macular edema (DME). ABBV-RGX-314 Suprachoroidal Delivery for Treatment of DR Based on positive interim results from the Phase II ALTITUDE® trial to date, the design and evaluation of two pivotal trials is on-going. With AbbVie, REGENXBIO has accelerated a planned EOP2 meeting with the FDA, now expected in Q4 2024. The Company expects to initiate the first global pivotal trial in the first half of 2025. The ALTITUDE trial is now enrolling a new cohort of patients with center-involved diabetic macular edema (DME). Patients will receive a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops. DME is a vision-threatening complication of diabetic retinopathy; an estimated 34 million people globally have DME. ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet AMD Enrollment is on track in the ATMOSPHERE® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the FDA and European Medicines Agency in the first half of 2026. The open label fellow eye study evaluating ABBV-RGX-314 in patients treated in the subretinal Phase I/IIa study is now fully enrolled. This study is designed to monitor safety, immune responses, and efficacy of ABBV-RGX-314 treatment in the fellow eye and these study data are intended to support the inclusion of bilateral use in the product label. Bilateral disease impacts a significant number of patients with wet AMD. ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD As of July 29, 2024, ABBV-RGX-314 at dose level 3 with short course prophylactic steroid eye drops continues to be well tolerated with no drug-related SAEs and no cases of intraocular inflammation, endophthalmitis, vasculitis, retinal artery occlusion, choroidal effusion, or hypotony. Mild episcleritis occurred in 3 patients, all resolved and completed treatment with topical steroids. There were no cases of elevated intraocular pressure. Based on this favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase II AAVIATE® trial is initiating enrollment in a new cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort will also receive short course prophylactic steroid eye drops. Neurodegenerative Disease: RGX-121 is a potential one-time AAV Therapeutic for the treatment of boys with MPS II. REGENXBIO completed a successful pre-BLA meeting with the FDA and will initiate a rolling BLA submission using the accelerated approval pathway in Q3 2024, potentially making RGX-121 the first approved gene therapy and one-time treatment for MPS II. REGENXBIO expects to share additional safety and efficacy data from the Phase I/II/III CAMPSIITE® trial in the second half of 2024. Approval of the planned BLA could result in receipt of a Priority Review Voucher in 2025. FINANCIAL RESULTS Cash Position: Cash, cash equivalents and marketable securities were $327.3 million as of June 30, 2024, compared to $314.1 million as of December 31, 2023. The increase was primarily attributable to $131.1 million of aggregate net proceeds received from the follow-on public offering of the Company's common stock and pre-funded warrants completed in March 2024, and was partially offset by cash used to fund operating activities during the first half of 2024. Revenues: Revenues were $22.3 million for the three months ended June 30, 2024, compared to $20.0 million for the three months ended June 30, 2023. The increase was primarily attributable to Zolgensma® royalty revenues, which increased from $19.0 million for the second quarter of 2023 to $21.8 million for the second quarter of 2024. Research and Development Expenses: Research and development expenses were $48.9 million for the three months ended June 30, 2024, compared to $59.9 million for the three months ended June 30, 2023. The decrease was largely driven by manufacturing and clinical supply costs for ABBV-RGX-314 and RGX-202, and personnel-related costs as a result of reduced headcount. The decrease was partially offset by increases in clinical trial expenses for ABBV-RGX-314 and RGX-202. General and Administrative Expenses: General and administrative expenses were $18.9 million for the three months ended June 30, 2024, compared to $23.7 million for the three months ended June 30, 2023. The decrease was primarily attributable to expenses for professional services and other corporate overhead costs. Net Loss: Net loss was $53.0 million, or $1.05 basic and diluted net loss per share, for the three months ended June 30, 2024, compared to a net loss of $72.1 million, or $1.66 basic and diluted net loss per share, for the three months ended June 30, 2023. FINANCIAL GUIDANCE REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $327.3 million as of June 30, 2024 to fund its operations into 2026. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any payments that may be received from AbbVie upon the achievement of development or commercial milestones under our ABBV-RGX-314 collaboration (including a potential, one-time $200.0 million milestone for achievement of first patient dosed in the first pivotal trial for suprachoroidal delivery for treatment of DR) and the potential monetization of a priority review voucher that may be received for RGX-121. CONFERENCE CALL In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. To access the live call by phone, dial (646) 307-1963 (domestic) or (800) 715-9871 (international) and enter the passcode 4849384. To access a live or recorded webcast of the call, please visit the Investors section of the REGENXBIO website at . The recorded webcast will be available for approximately 30 days following the call. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA® for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie, the monetization of any priority review voucher, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2023, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO. CONTACTS: Dana Cormack Corporate Communications [email protected] Chris Brinzey, ICR Westwicke 339-970-2843 [email protected] SOURCE REGENXBIO Inc.