Gene Therapy Updates at ASRS Provide Incremental Support for Pivotal Dose Levels

Research evidence excerpt

Gene Therapy Updates at ASRS Provide Incremental Support for Pivotal Dose Levels

Update

July 20, 2026 04:01 AM GMT

Morgan Stanley & Co. LLCMBiotechnology | North America Judah C. Frommer, CFA

Equity Analyst

Gene Therapy Updates at ASRS Judah.Frommer@morganstanley.comNicholas D Japhet +1 212 761-1270

Research Associate

Nick.Japhet@morganstanley.com +1 212 761-2721

Provide Incremental Support for Parth Patel

Parth.Patel@morganstanley.com +1 212 761-5065

Pivotal Dose Levels

Biotechnology

North America

Industry View Attractive

Key Takeaways

FDMT and RGNX reported longer-term data at ASRS from Ph1/2 studies of their

gene therapies in wet AMD (4D-150 and RGX-314) and DR (RGX-314).

The data provided incremental support for the durability/safety of dose levels

selected for pivotal development.

Topline Ph3 data in wet AMD are expected for RGX-314 in 4Q26 and 1H27 and

2H27 for 4D-150.

FDMT and RGNX presented longer-term follow-up data from Ph1/2 studies of

their anti-VEGF gene therapies (4D-150 and RGX-314, respectively) in wet AMD

(and in DR for RGX-314) at ASRS. The updates further informed the clinical profiles

of the pivotal doses currently being evaluated in ongoing Ph3 wet AMD studies and

RGNX's recently initiated Ph2b/3 NAAVIGATE study in DR. Though sample sizes

from the studies are relatively limited and the data can be challenging to interpret in

some ways (e.g., due to differences in supplemental injection criteria/practices for

wet AMD), the updates provided incremental support for the durability and safety

of 4D-150 and RGX-314, in our view. The lack of late-presenting, treatment-related

IOI events among patients treated at the pivotal doses should provide incremental

comfort to retina specialists worried about the potential risk of gene therapies and