Core Viewpoint - REGENXBIO Inc. has announced that the FDA has placed a clinical hold on its investigational gene therapies RGX-111 and RGX-121 due to a case of neoplasm identified in a participant treated with RGX-111, raising concerns about the safety of both therapies [1][2]. Group 1: Clinical Hold Details - The FDA's clinical hold on RGX-111 and RGX-121 is based on a single case of an intraventricular CNS tumor found in a five-year-old participant who received RGX-111 four years prior [1][2]. - Preliminary genetic analysis of the tumor indicated an AAV vector genome integration event linked to overexpression of the proto-oncogene PLAG1, which is associated with chromosomal rearrangements [2]. Group 2: Company Response - REGENXBIO expressed surprise at the FDA's decision to place RGX-121 on hold, emphasizing that RGX-121 has a positive safety profile based on data from over 30 patients treated [3]. - The company highlighted the urgent medical need for RGX-121 in treating MPS II, stating that delays could lead to neurodevelopmental decline in affected patients [3]. Group 3: Therapy Information - RGX-121 is a one-time gene therapy designed to deliver the iduronate-2-sulfatase (IDS) gene to the CNS, potentially providing a permanent source of the I2S protein beyond the blood-brain barrier [4]. - RGX-111 aims to deliver the alpha-L-iduronidase (IDUA) gene to the CNS, which could help prevent cognitive deficits in MPS I patients [7]. Group 4: Disease Background - MPS II, or Hunter Syndrome, is a rare disease caused by a deficiency in the lysosomal enzyme I2S, leading to the accumulation of glycosaminoglycans and resulting in dysfunction across various tissues, including the CNS [6]. - MPS I is a rare genetic disease caused by a deficiency in the enzyme IDUA, leading to similar accumulations and dysfunctions, with an estimated occurrence of 1 in 100,000 births [9].

Core Viewpoint - REGENXBIO Inc. will present at the 44th Annual J.P. Morgan Healthcare Conference, highlighting its advancements in gene therapy and its late-stage pipeline of treatments for rare and retinal diseases [1][2]. Company Overview - REGENXBIO is a biotechnology company founded in 2009, focused on gene therapy with a pioneering role in AAV gene therapy [3]. - The company is advancing a late-stage pipeline that includes treatments for Duchenne (RGX-202), MPS II (clemidsogene lanparvovec, RGX-121), and MPS I (RGX-111), in partnership with Nippon Shinyaku [3]. - Additionally, REGENXBIO is collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3]. - Thousands of patients have been treated using REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3]. - The investigational gene therapies from REGENXBIO have the potential to significantly impact healthcare delivery for millions [3].

Core Insights - REGENXBIO Inc. will participate in the Piper Sandler 37th Annual Healthcare Conference with a fireside chat scheduled for December 2, 2025, at 8:30 a.m. ET [1] - The company is focused on advancing gene therapy, particularly in treating rare and retinal diseases, with a late-stage pipeline that includes several investigational therapies [2] Company Overview - REGENXBIO, founded in 2009, specializes in AAV gene therapy and aims to improve lives through its curative potential [2] - The company has developed treatments such as RGX-202 for Duchenne muscular dystrophy, RGX-121 for MPS II, and RGX-111 for MPS I, in collaboration with Nippon Shinyaku [2] - REGENXBIO is also working with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [2] - Thousands of patients have been treated using REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [2] Recent Developments - The company announced the completion of pivotal enrollment in the AFFINITY DUCHENNE® trial for RGX-202, marking a significant milestone in its Duchenne gene therapy program [5]

Core Insights - REGENXBIO Inc. announced the presentation of interim data from the Phase II ALTITUDE trial for surabgene lomparvovec (ABBV-RGX-314) targeting diabetic retinopathy at the American Academy of Ophthalmology 2025 Annual Meeting [1][2] - Surabgene lomparvovec is a one-time gene therapy developed in collaboration with AbbVie, aimed at treating wet age-related macular degeneration and diabetic retinopathy [1][2] Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, founded in 2009, and has developed a late-stage pipeline for various diseases, including RGX-202 for Duchenne and RGX-121 for MPS II [3] - The company utilizes the NAV AAV8 vector in surabgene lomparvovec, which encodes an antibody fragment to inhibit vascular endothelial growth factor (VEGF), a key factor in the development of leaky blood vessels in the retina [2][3]

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on August 7, 2025, at 8:00 a.m. ET to discuss its financial results for Q2 2025 and operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session by dialing specific numbers [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]

Core Viewpoint - REGENXBIO Inc. has secured a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty, which will enhance its cash runway and support upcoming milestones in its gene therapy pipeline [1][2][3] Financing Details - The agreement allows REGENXBIO to receive $150 million at closing, with an extension of cash runway into early 2027 [1][8] - An additional $50 million will be funded upon achieving sales milestones for ZOLGENSMA, and another $50 million will be available upon mutual agreement between the parties [5][6] Strategic Implications - This financing is aimed at advancing late-stage activities, including potential FDA approvals and data readouts for key products such as RGX-121 and RGX-202 [2][3] - The capital infusion is expected to accelerate commercial preparations and maintain REGENXBIO's leadership in rare and retinal gene therapies [2][8] Agreement Terms - HCRx will receive rights to anticipated royalty payments from ZOLGENSMA and other products, with quarterly interest payments based on royalty and milestone revenue [3][4] - The agreement does not include other potential non-dilutive funding sources, such as the sale of a Priority Review Voucher for RGX-121 [6][8] Company Background - REGENXBIO is focused on gene therapy, with a late-stage pipeline targeting rare and retinal diseases, including partnerships with Nippon Shinyaku and AbbVie [8][9] - The company has pioneered AAV gene therapy and aims to improve healthcare delivery through its investigational therapies [9]

Company Overview - REGENXBIO Inc. is a biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has been a pioneer in AAV gene therapy since its founding in 2009 [3] - REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-111 for MPS I, in partnership with Nippon Shinyaku [3] - The company is also collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3] Upcoming Events - REGENXBIO will host a conference call on May 12, 2025, at 4:30 p.m. ET to discuss its financial results for Q1 2025 and recent operational highlights [1] - Listeners can register for the webcast and analysts can participate in the Q&A session through provided links [2] - A replay of the webcast will be available on the company's investor website approximately two hours after the call [2]