Executive Vice President & Chief Medical Officer, Regenxbio
Search every verified Stephen Pakola interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Stephen Pakola, Executive Vice President and Chief Medical Officer at Regenxbio, has been discussing the company's progress across multiple gene therapy programs in 2024 and 2025. He stated that Regenxbio is on the cusp of potential commercialization for three lead assets, including RGX-121 for Hunter syndrome (MPS II), for which he said the company anticipated finishing a Biologics License Application submission and receiving approval under accelerated approval later in 2025. Pakola also highlighted the RGX-314 program for retinal conditions, which he described as the largest gene therapy program ever conducted, with global pivotal trials enrolling 1,200 patients. He noted that the company and its partner AbbVie were advancing plans for a pivotal program in diabetic retinopathy based on Phase 2 data showing an 89% reduction in vision-threatening complications at a higher dose level. Pakola has emphasized Regenxbio's shift from licensing its NAV vector platform to advancing its own internal pipeline, including programs for MPS I and MPS II, Duchenne muscular dystrophy, and wet age-related macular degeneration. He has discussed the company's use of suspension cell bioreactor production to improve manufacturing yields and purity, and has pointed to data from MPS trials showing biomarker reductions and developmental improvements in treated children. Pakola has also described the company's strategy of using both subretinal and suprachoroidal delivery routes for RGX-314, with the latter intended to enable in-office administration for diabetic retinopathy patients.
“We at Regenex Bio our mission is to improve lives through the curative potential of our NAV platform of AAV vectors.”
“One of the significant milestones in the whole field of rare disease and gene therapy of course was Zolgensma for the treatment of spinal muscular atrophy, which is based on our NAV vector AAV9 to deliver that transgene.”
“We initially as a company were focused on delivering our NAV platforms like AAV9 and AAV8 to licensees who would advance them the way Novartis has, but over the last five years or so we've transitioned our priority to advancing programs ourselves.”
“MPS1 and MPS2 are monogenic lysosomal storage disorders where patients are missing particular enzymes, leading to significant buildup of glycosaminoglycans—particularly heparan sulfate—which is correlated with neurocognitive and neurofunctional decline.”
Steve Pakola, MD, Chief Medical Officer for Regenxbio, discusses data from the ongoing gene therapy trials in children with ...
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REGENXBIO is a longtime player in the gene therapy space, but one that's yet to bring its own therapy to market (in the early days ...
... lives through the Curative potential of gene therapy and we seek to do this with our proprietary set of a vectors from what we call ...
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... down with Dr. Steve Pakola, at OIS@ASRS 2019. REGISTER for our next ophthalmology conference today http://bit.ly/2yFA6cv.
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