JP Morgan Week 2025 – Steve Pakola

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 it focused on licensing its NAV Technology platform to other companies.)

But all that could be about to change, as the company has three assets in its pipeline that could make it to market in the next one to three years. On the sidelines of JP Morgan in San Francisco, editor-in-chief Jonah Comstock caught up with Steven Pakola, REGENXBIO’s chief medical officer, to hear a little bit about each of those three assets – RGX121 for Hunter’s Syndrome, RGX202 for Duchenne’s muscular dystrophy, and RGX134, focused on retinal conditions in partnership with AbbVie.

Pakola also discusses some of the difficulties inherent in developing gene therapies and how REGENXBIO’s decade-plus of experience in the space has helped position it to overcome those challenges.

Tune in for an update on three exciting upcoming gene therapies and a discussion of the gene therapy landscape more broadly.