Phase 3 clinical trial has been given approval by FDA
The FDA has given Ocugen permission to start their phase 3 gene therapy clinical trial for retinitis pigmentosa (RP).
Biotechnology company Ocugen has announced they have received clearance from the FDA for their phase 3 clinical trial of OCU400. OCU400 is a gene-agnostic therapy for retinitis pigmentosa (RP), which could be appropriate for a large proportion of our community no matter what their genetic diagnosis.
OCU400 is based on a gene called NR2E3, which regulates various physiological functions within the retina like photoreceptor development and maintenance, metabolism, inflammation and cell survival networks. It works by resetting the affected cellular networks in the retina to establish a state of healthy balance (homeostasis). The treatment has the potential to improve retina health and function in people with RP.
OCU400 will be the first gene-agnostic gene therapy product to enter the phase 3 study stage. The study will involve 150 participants split into two groups. The first group will include 75 participants with RP caused by mutations in the RHO gene. The other 75 participants will be the “gene agnostic” group, meaning that these participants have various different genetic diagnoses or possibly do not know the gene causing their RP. Within each group, one third of the participants will be untreated and act as controls.
Ocugen is also undertaking an earlier phase I/II trial in Stargardt disease.
Both trials will take place across sites in the USA. These centres will select patients who fit the genetic diagnosis and numerous other criteria. For more information, contact clinical.request@ocugen.com.