On Monday, it was announced that Ocugen, Inc. (OCGN) has received approval from the FDA to initiate a Phase 3 clinical trial for their Investigational New Drug or IND, OCU400.
OCU400 is a pioneering gene therapy product being developed to treat retinitis pigmentosa - a group of rare genetic disorders known for causing progressive vision loss and blindness.
The impending Phase 3 trial will involve around 150 participants split into two groups. The first group will consist of 75 participants with the RHO gene mutation, while the second group will include 75 gene-agnostic participants. Each group will be randomly assigned on a 2:1 basis to either receive a treatment dosage of 2.5 x 10^10 vg/eye of OCU400 or to be part of a control group not receiving the treatment.
Ocugen confirmed that this pivotal Phase 3 clinical trial puts OCU400 firmly on track for a projected Biologics License Application (BLA) approval by 2026.
