Regenxbio (RGNX) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Company overview and strategic focus

• Focused on gene therapy using AAV vectors, with programs in retina, CNS rare diseases, and Duchenne muscular dystrophy (DMD).

• In-house manufacturing capabilities in Rockville, Maryland, supporting late-stage development and commercial production.

• Pipeline includes RGX-202 for DMD, RGX-121 for Hunter syndrome, and RGX-314 for retinal diseases.

• Partnerships with AbbVie (retina) and MS Pharma (Hunter syndrome) expand commercial reach.

• Monetization strategies include royalty agreements and leveraging priority review vouchers.

RGX-202 for Duchenne muscular dystrophy

• Differentiated by inclusion of the C-terminus in microdystrophin construct and high-purity vector manufacturing.

• Unique immune suppression regimen has resulted in a strong safety profile with no significant adverse events.

• Robust microdystrophin expression and functional improvements observed, including in older patients.

• Enrollment for pivotal trial targeted for completion in October, with commercial launch aimed for 2027.

• Growing market opportunity as delays in competitors' products increase addressable population.

RGX-121 for Hunter syndrome

• MS Pharma to commercialize in US and Asia, with double-digit royalties and a bond structure reverting royalties after payoff.

• FDA requested 12-month data, which was provided and showed consistent positive results, strengthening the approval package.

• BLA review progressing well, with no major observations from FDA inspections.

• Commercial launch readiness supported by completed production campaigns and payer engagement.