RegenxBio's AMD gene therapy well tolerated in Phase I

RegenxBio Inc. (NASDAQ:RGNX) said a single subretinal injection of RGX-314 was well tolerated with no treatment-related serious adverse events reported in a Phase I trial to treat wet age-related macular degeneration (AMD). RGX-314 is an adeno-associated virus serotype 8 (AAV8) vector carrying a gene for a mAb fragment that binds to VEGF and neutralizes VEGF activity.

The open-label, dose-escalation, U.S. trial enrolled 18 patients who responded to previous anti-VEGF therapy to receive one of three dose levels of a single subretinal injection of RGX-314...