Company: RGNX
Filing Date: 2025-04-08
Form Type: DEF 14A
Source: 0000950170-25-052069
Chunk: 4

Company: REGENXBIO Inc.
Filing Date: 2025-04-08
Form: DEF 14A
Chunk 4
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 lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people.

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Business Highlights In 2024, we made significant progress toward our clinical development and business objectives, including the following achievements:

| ABBV-RGX-314 for the Treatment of wet AMD and DR under the eyecare collaboration with AbbVie | We achieved key enrollment milestones with ABBV-RGX-314 ATMOSPHERE® and ASCENT™ pivotal clinical trials evaluating the efficacy and safety of ABBV-RGX-314 in patients with wet AMD using the subretinal delivery approach. We announced positive new data from multiple trials in the ABBV-RGX-314 program, including from the Phase II bridging study evaluating the clinical performance using the NAVXpress™ manufacturing platform process and the phase II AAVIATE® trial for the treatment of wet AMD using suprachoroidal delivery and the phase II ALTITUDE® trial using in-office suprachoroidal delivery for the treatment of DR without center-involved diabetic macular edema (DME). We completed a successful End-of-Phase II meeting with the FDA for our Phase II ALTITUDE trial and expanded enrollment into a new cohort of patients with center-involved DME.                                                                                                                           |
| RGX-202 for the Treatment of Duchenne Muscular Dystrophy (“Duchenne”)                        | We delivered on RGX-202 milestones, including a successful End-of-Phase II meeting with the FDA, initiating a pivotal (Phase III) trial of AFFINITY DUCHENNE®, approval for trial initiation in Canada, and announcing first strength and functional assessment data for both dose levels.                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                 |
| RGX-121 for the Treatment of Mucopolysaccharidosis Type II (“MPS II”)                        | We announced that the pivotal phase of the CAMPSIITE® trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with MPS II achieved its primary endpoint in addition to reporting positive long-term safety and tolerability data.We also announced a successful pre-Biologics License Application (BLA) meeting for RGX-121, where we finalized details of our BLA with the FDA. Then in alignment with the FDA we completed