Itvisma: how long were the pivotal tox studies?

“These studies demonstrated that OAV101 was well tolerated at this dose level up to 12 months post injection, with minor microscopic findings reported 6 weeks post-administration in the dorsal root ganglia (DRG), spinal cord, peripheral nerves, and brain that did not increase in severity or incidence over the 12 month study.”

“A single IT dose of onasemnogene abeparvovec was administered at three dose levels: 1.20 × 10 13 vg/animal (equivalent to the clinical therapeutic dose), 3.0 × 10 13 vg/animal, and 6.0 × 10 13 vg/animal.”

“Additional single-dose studies in the newborn Friend Virus B (FVB) mouse strain and NHPs further demonstrated high tolerability to OAV101 or scAAV9.CBA.SMN following ICV or IT administration, respectively, with no major safety signals.”